Category Archives: Gene Therapy Clinics


Endothelial Dysfunction Industry 2019-2025 Market Analysis by Size, Share, Growth, Regional, Manufactures, Application and Forecast Research Report -…

The Asia-Pacific Endothelial Dysfunction market was valued at USD XX million in 2017, and is expected to grow at a CAGR of XX% by 2025. Endothelium dysfunction can be defined as partial or complete loss of balance between vasodilating and vasoconstricting substances produced by the endothelium.

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The Global Endothelial Dysfunction Industry based on geographic classification is studied for industry analysis, size, share, growth, trends, segment, top company analysis, outlook, manufacturing cost structure, capacity, supplier and forecast to 2025. Along with the reports on the global aspect, these reports cater regional aspects as well as global for the organizations

Top Key Vendors:

Abbott LaboratoriesChugai Pharmaceutical Co. Ltd.Johnson & JohnsonGlucox Biotech ABStealth Peptides Inc

Rise in number of obese populations, reduced physical activities and diabetes leading to increase in chances of acquiring cardiovascular diseases will propel the market growth in the forecast period. Stringent regulation regarding the drug will hamper the market growth in the forecast period. Advances in recombinant and gene therapy are also favoring the growth of endothelial dysfunction drugs uptake and can be an opportunity area for new entrants in the market.

Based on application, the market is segmented into, hospital, clinics among others.

Based on diagnosis, the market is segmented into, coronary circulation, angiography of coronary artery.

Based on region the market is segmented into China, India, Japan, South Korea, Taiwan, Southeast Asia, and Australia.

Global Endothelial Dysfunction Industry is spread across 121 pages, profiling 05 companies and supported with tables and figures.

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Key Benefits of the Report:* Global, Regional, Country, Learning Module, and Type Market Size and Forecast from 2014-2025-Provide attractive market segments and associated growth opportunities* Detailed market dynamics, industry outlook with market specific PESTLE, Value Chain, Supply Chain, and SWOT Analysis to better understand the market and build strategies* Identification of key companies that can influence this market on a global and regional scale* Expert interviews and their insights on market shift, current and future outlook and factors impacting vendors short term and long term strategies* Detailed insights on emerging regions, product & Learning Module, and competitive landscape with qualitative and quantitative information and facts

Target Audience:* Endothelial Dysfunction providers* Traders, Importer and Exporter* Raw material suppliers and distributors* Research and consulting firms* Government and research organizations* Associations and industry bodies.

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TABLE OF CONTENT

Introduction

Methodology & Scope

Global Endothelial Dysfunction Market Overview

Asia-Pacific Endothelial Dysfunction Market by Diagnosis type

Asia-Pacific Endothelial Dysfunction Market by Application

Asia-Pacific Endothelial Dysfunction Market by Region

Competitive Landscape

Company Profiles

End of TOC

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Endothelial Dysfunction Industry 2019-2025 Market Analysis by Size, Share, Growth, Regional, Manufactures, Application and Forecast Research Report -...

Cell Harvesting Market is Expected to Reach USD 387.9 Million by 2025| Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical, Argos Technologies -…

The Global Cell Harvesting Market is expected to reach USD 387.9 Million by 2025, from USD 196.9 Million in 2017 growing at a CAGR of 8.9% during the forecast period of 2018 to 2025. Some of the major players operating in the global cell harvesting market are PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical, Argos Technologies, SP Industries, Teleflex Incorporated, Arthrex, Inc, Thomas Scientific, Brand GMBH, Brandel, Cox Scientific, Connectorate, Scinomix, Adstec.

Global Cell Harvesting Market,By Type (Manual Cell Harvesters and Automated Cell Harvesters), By Application (Biopharmaceutical Application, Stem Cell Research and other Applications), By End Users (Hospitals, Ambulatory Centers, Clinics, Community Healthcare, Others), By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2025

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Market Definition:Global Cell Harvesting Market

This market report defines the market trends and forecast the upcoming opportunities and threats of the cell harvesting market in the next 8 years. Cell harvesting is extracting the cells either from bone marrow and peripheral blood cells and culturing the cells in the culture dish containing nutrient media. Cell harvesting is used in the cell therapy as well as in gene therapy. University of California developed a cure for bubble baby disease for new born babies by using the cell harvesting in stem cells and gene therapy. Moreover, Asterias developed the stem cell therapy to regain the upper body motor function. University of California, Irvine developed the stem cell therapy to destroy the breast cancer cells.. Now a days cell harvesting is also used in the animal research and development. Cell Harvesting is also used in may research labs for in-Vitro testing. In September 2016, Terumo BCT collaborated with Cognate Bioservices for developing the immunotherapies and other related products like cell therapy products. These innovations in the cell harvesting market is notably attributing towards its increasing demand at the global pace. Further, its demand is likely to gain momentum over the forecast period.

Major Market Drivers and Restraints:

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Market Segmentation:Global Cell Harvesting Market

The global cell harvesting market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cell harvesting market for global, Europe, North America, Asia Pacific and South America.

Research Methodology:Global Cell Harvesting Market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analyzed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more pleaseRequest an Analyst Callor can drop down your inquiry.

Demand Side Primary Contributors: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.

Supply Side Primary Contributors: Product Managers, Marketing Managers, C-Level Executives, Distributors, Market Intelligence, Regulatory Affairs Managers among others

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Cell Harvesting Market is Expected to Reach USD 387.9 Million by 2025| Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical, Argos Technologies -...

Insights on precision oncology developments in Japan and beyond – MobiHealthNews

Cancer has been the leading cause of death in Japan since 1981 and this is compounded by the fact that the country has a hyper-aging society, which means that Japan will face a substantial increase in the number of elderly cancer patients, according to a review article by Matsuda and Saika published in the Annals of Cancer Epidemiology in 2018. Prof Yasushi Goto of the National Cancer Center Hospital in Japan shared with MobiHealthNews on some of the latest developments in precision oncology in Japan, the interest of pharmaceutical companies in targeted therapy and a nationwide cancer genome screening project named SCRUM.

Q. Could you tell us more about your role at the National Cancer Center Hospital?

A. National Cancer Center Hospital (NCCH) is based in Tokyo, Japan and is the largest cancer center in the country. The other is national cancer center is located in Chiba, Japan. One of the special characteristics of NCCH apart from the research institute hospital, is that we also do research for every kind of cancer activity in our hospital. We dont only do clinics, but also genetic oncology, rare cancers.

My main work is at thoracic oncology which mainly covers lung cancer. I also am a member of Rare Cancer Center so I also cover, malignant mesothelioma, thymic cancer, neuroendocrine tumors, and all others. Since Japan has launched national genomic project, and National Cancer Center is playing the central role in this project, I am also joining Section of Knowledge Integration in Center for Cancer Genomics and Advanced Therapeutics.

Q. What are your observations on the development of precision oncology in the APAC region? How would you describe precision oncology in the most straightforward manner?

A. Testing for patients with cancer is prevalent in the APAC region. Compared to other countries, people in Asia are generally accustomed to genetic testing for precision medicine. Our capabilities may not be as advanced as the United States for full gene sequencing, perhaps because of cost issues, but we are catching up.

For precision oncology to develop further, more patients should be tested for genetic testing and target therapy. We are looking to putting in place a proper system to support this right now, especially in the rare cancer field.

There was a recent nationwide project in Japan called SCRUM, conducted by the National Cancer Center East Hospital. SCRUM is the first industry-academia collaboration nationwide cancer genome screening project. I believe early access to drugs is partly due to this project.

Q. Medtech or healthtech startups are also getting into the precision medicine space and seeing a lot of interest from investors. For instance, genomic medicine startup Lucence from Singapore recently raised $20 million in Series A funding. What opportunities do you see in these startups in terms of working together with hospitals such as the National Cancer Centre Hospital? Are there any notable startups in Japan in the precision medicine space?

A. In Japan, many pharmaceutical companies are looking at targeted therapy. Prominent companies include Daichi-Sankyo, Chugai, and Takeda. NCCH is currently working closely with Daichi-Sankyo, Takeda and Chugai.

The approval system is different between blockbuster drugs and targeted therapy. If you only have one patient in Japan and globally there are only 20 or so there can be a scenario where the drug is effective, but it is not approved in our country as there is only a single patient. This is why some pharmaceutical companies are looking at precision medicine.

Companies including Sysmex are also actively looking into the testing of panel sequencing. NCCH has also worked with Sysmex to make a cancer sequencing panel.

Q. The high incidence of cancer in modern societies is worrying and also very costly how do you think precision oncology can help tackle some of these challenges?

A. Cost is a global issue, for both the development side (e.g. the pharmaceutical companies) and the consumption side (e.g. governments, individuals). We also need some basic infrastructure for precision oncology, to screen patients. Currently each drug needs its own testing. With many drugs that need to be tested, we need a platform to do panel sequencing in order to annotate any genetic changes in the patients for treatment.

In Japan, we are now trying to make this infrastructure because the government recently approved the panel sequencing in 2019. This means that after standard therapy, every patient is able to test for panel sequencing. As more patients are screened, in this way I think cancer treatment will be improved in the future.

Q. What do you think will be the key developments and breakthroughs in precision oncology in the next 3-5 years?

A. There will be no blockbuster development, but there will be steady progress in the detecting genetic changes earlier in the next 3-5 years. Some of the topics at the ESMO Asia Congress 2019 in Singapore were on advancements in detecting mutations/previously overlooked genes. Since we are able to find these abnormalities earlier, we now have new anti-cancer agents to target them.

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Insights on precision oncology developments in Japan and beyond - MobiHealthNews

Novel discovery in gene therapy to treat kidney diseases – WeForNews

Jaipur, Jan 4 : The sorry state of Kotas J.K. Lon Hospital is reflected in a government-appointed panels report which shows that around 105 children died in a span of over a month due to cold shivering in the hospital as it lacked everything a normal hospital should have.

The committee formed by the Rajasthan government to probe the lacunae in the hospital resulting in the deaths of kids has confirmed in its report that infants died due to hypothermia, a medical emergency that occurs when the body temperature falls below 95 F (35 C). The normal body temperature is 98.6 F (37 C).

Even as the kids in the hospital continued to die in the biting winter cold, the hospital did not have enough stocks of lifesaving equipment, said the report.

The newborns should have body temperature of 36.5 degree celcius; therefore they were kept on warmers where their temperature stays normal. However, as the hospital lacked functional warmers, their body temperature continued to plummet.

The report said that 22 nebulisers out of 28 were dysfunctional, 81 infusion pumps out of 111 were not working and the same was the story with para monitors and pulse oxymeters.

What made the matters worse was the absence of oxygen pipeline in the hospital due to which oxygen was supplied to kids with the help of cylinders.

Surprisingly, the ICU was not fumigated for months, the report said.

The children continued to die in December as Chief Minister Ashok Gehlot celebrated the launch of Nirogi Rajasthan campaign in the state, said former health minister Rajendra Rathore.

Hospital officials said most paediatricians of JK Lon Hospital have been posted at Kotas New Medical College. The nursing employees, already under-staffed, prefer to stay idle while ward boys rule the roost in the J.K. Lon Hospital, said an official.

Also, the hospital staff continued with the whitewash in wards where infants suffering from pneumonia were admitted for oxygen.

According to sources, there was no record of 40 heaters purchased for kids. A hospital official said despite Rs 6-crore funds lying with the hospital, no purchase has been made.

On Friday, when state Health Minister Raghu Sharma visited the hospital, a green carpet welcome was given to him, a gesture hardly suited for the grim situation prevailing in the hospital. After drawing severe criticisms, the carpet was rolled back.

A five-month-old girl suffering from pneumonia died the same day when Sharma visited the hospital as the officials were busy getting the walls cleaned.

Former Health Minister Rajendra Rathore said rampant transfer of specialists on political grounds has deprived the patients of availing the best medical services.

Rathore said that during BJPs rule, we ensured that all specialists were kept at one place irrespective of any political leaning.

He said the present government has not had annual repair contracts of the equipment purchased under its tenure. There is no medical inspection arrangement for the equipment purchased, Rathore said.

Gehlot in December celebrated one year of the formation of his government and launched the Nirogi Rajasthan campaign. He also announced Janata Clinics and spoke about introducing Right to Health.

How can Right to Health be launched when there is no homework done by the state government? questioned Rathore.

The Nirogi campaign is only on papers as there has been no discussion on it with panchayats or other stakeholders, Rathore said. In a situation when 11,000 posts of doctors are lying vacant, how can the government talk about Janata clinics? he asked.

State Health Minister Raghu Sharma when contacted by IANS was not available for comment.

(Archana Sharma can be contacted at [emailprotected])

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Novel discovery in gene therapy to treat kidney diseases - WeForNews

Top Providers of the Alpha Thalassemia Market gives a New Dimension easing Measurement and Quantification – GuruFocus.com

Inclusive study on Alpha Thalassemia Market to Attractive Growth by Strategic Industry Evolutionary Analysis 2029| Segmentation, Future Scope, Development Strategies, Competitive Breakdown, and Regional Forecast. The market is expected to remain prominent in the Asia Pacific, Center East, and Africa, North America, South America, and Europe regions as companies are intensifying business operations and focusing on increasing market share.

Explosive Growth In Alpha Thalassemia Market size in terms of revenue (USD MN) is calculated for the study along with the aspects of the factors affecting the market growth (drivers and restraints). The market for alpha thalassemia has been categorized into various segments, for analysis, on the basis of the type, application area, and region. Detailed analysis of alpha thalassemia market Predicted to grow by 2029 with competitive Analysis identifying the key mutual trends and major players of the market and business guidelines till 2029. This strong growth outlook of the Alpha Thalassemia market has been attributed to the advancements in computing capacity for Alpha Thalassemia-based research and applications.

Furthermore, the report offers an in-depth understanding of alpha thalassemia market-particular drivers, constraints and major micro markets. The report highlights many significant points and trends of the industry which are helpful for our esteemed clients. In this research study, the manufacturing base, productivity, manufacturers, strengths, recent trends, features are identified in detailed.

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[Note: Our Free Complimentary Sample Report Accommodate a Brief Introduction To The Synopsis, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology]

About Alpha Thalassemia Industry

As per the findings of a new Market.us report titled,Global Alpha Thalassemia Market By Type (Iron Chelating Drugs, Gene Therapy),By Application (Hospitals, Private clinics) and by Region Global Forecast to 2029.This offers a holistic view of the alpha thalassemia market through systematic segmentation that covers every aspect of the target market. The study provides valued information on key competition trends and the most preferred developmental strategies of leading market brands. The analysis covers alpha thalassemia market and its advancements across different industry verticals as well as regions. It targets estimating the current market size and growth potential of the alpha thalassemia industry across sections such as also application and representatives.

Additionally, the analysis also has a comprehensive review of the crucial players on the alpha thalassemia market together side their company profiles, SWOT analysis, latest advancements, and business plans.

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For the Alpha Thalassemia Market research study, the following years have been considered to estimate the market size:

Historic Year:2012 to 2018

Estimated Year:2019

Forecast Year:2020 to 2029

The Alpha Thalassemia Market report serves as a vital guide in portraying present and forecast industry statistics

-The supply/ demand situation, gross margin view and competitive profile of top Alpha Thalassemia players (Bluebird Bio, Novartis, Kiadis Pharma) are presented.

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This was the best health news over the last decade – USA TODAY

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The 2010s will go down in history as a decade of many newsworthy health-related stories, many of which were not good news -- Ebola, measles, antibiotic resistance. But in the years since 2010 there were also many promising discoveries in medicine, life-saving drugs approved, and great strides taken addressing national health crises. Some of these stories will have lasting effects for generations to come.

24/7 Tempo reviewed multiple news archives and dozens of articles published since 2010 to select 15 of the most positive health news stories that made headlines.

Some of the most talked about stories over the last few years have influenced health guidelines, treatment of serious disease, and even government policy.

Reports of significant research developments in the treatment and prevention of chronic and other conditions gave hope to millions of Americans. Some of the good news broke as recently as just a few months ago these are the 15 biggest health topics of 2019.

Click here for 15 of the best health news over the last decade.

CT scans in high risk patients can reduce overall lung cancer mortality

Year: 2011

Category: Diseases

The tremendous effort by researchers and health institutions to develop a cure for cancer over the decades since the legislation for the War on Cancer was enacted in 1971 will likely continue. Any good news on developments are worth noting. The 2011 National Lung Screening Trial showed a reduction in lung cancer mortality of 20% in high risk patients receiving low-dose CT (LDCT) compared to chest X-ray. The CDC recommends that people at high risk of developing lung cancer -- heavy smokers, people who have smoked as recently as 15 years, and people who are 55 years or older -- undergo annual LDCT scans because of potential risks.

In 2016, there were 218,229 new cases of lung cancer, and 148,869 people died from the disease in the United States, according to the CDC. The American Cancer Society estimates 142,670 deaths from lung cancer in 2019. A major reason for the disease's high mortality rate is that the tumor does not typically cause symptoms until it spreads, making early screening especially crucial to improving survival rates.

Blame SUVs: These 9 cars will be killed in 2020

Pass the ketchup, hold the beef: Americans crave Impossible Burger, Beyond Meat in 2020

Melanoma drug approved

Year: 2011

Category: Treatment

After more than a decade of no new potential drugs for melanoma, the deadliest form of skin cancer, the FDA approved vemurafenib, sold under the brand name Zelboraf, in 2011 for patients with metastatic melanoma with the BRAF(V600E) mutation or for those who have tumors that cannot be surgically removed.

Zelboraf was seen as a major development because it can improve melanoma patients' quality of life -- the drug is a simple pill taken twice a day -- and it may extend survival rate. In a trial, the length of time melanoma patients who received Zelboraf lived without the cancer getting worse was almost double the length of patients who did not take the drug.

Since 2011 several drugs have been approved to treat melanoma, and survival rates of this deadly cancer have improved.

Gene editing is now possible

Year: 2012

Category: Technology

Gene editing is the process of changing an organisms DNA. After decades of research around the world, scientists made a major breakthrough with the discovery of clustered repeats of DNA sequences, known as CRISPR.

First described in 2012, CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is the basis for potentially world-changing gene editing technology, or, as some might say, DNA hacking. It may be used to develop treatments for a range of diseases, including cancer and genetic disorders. In 2015, CRISPR was successfully used for the first time to save a life. Two baby girls, 11-month and a 16-month-old, received gene editing treatment to help them fight leukemia.

While the gene-altering tool is bringing revolutionary change to health fields, it has also raised serious ethical concerns. Misuses and inadvertently harmful uses of CRISPR include those for creating designer babies, and causing environmental ripple effects by eliminating disease-spreading insects.

FDA says trans fat should not be considered 'safe'

Year: 2013

Category: Eating

Trans fats, or partially hydrogenated oils, have been widely used for years, most notably in fast foods. Trans fats can raise the levels of "bad" LDL cholesterol, lower the levels of good-for-you HDL cholesterol, and increase the risk of heart disease, the No. 1 killer in the United States. And now they are on their way out.

In 2013, the FDA officially announced trans fats should not be considered safe in human food. In 2015, the agency gave food manufacturers three years to phase out the use of trans fats in their products. The deadline was June 18, 2018, although the FDA granted a one-year extension in the use of artificial trans fats in some cases. The ban will be fully implemented in Jan. 1, 2020.

HIV prevention pill

Year: 2014

Category: Diseases

About 50,000 Americans are diagnosed with HIV every year, according to the CDC. Despite advancements in treatment and years of research into the infection, HIV does not have a cure. In 2014, the CDC issued new guidelines that recommend a pill to people at high risk of HIV as a prevention method. High risk people include gay or bisexual men, injection drug users, and women with an HIV+ partner.

The agency said that the pill, sold under the brand name Truvada, may lower the risk by as much as 90% when taken consistently. Truvada has been used to treat HIV since 2012 when the FDA approved the drug. Truvada contains tenofovir and emtricitabine, which when used in combination with other antiviral medication may keep the HIV virus from establishing a permanent infection.

A new way to treat cavities

Year: 2015

Category: Treatment

In 2015, the FDA approved a painless new way to treat tooth decay called silver diamine fluoride (SDF). It's a liquid that is applied directly to cavities to stop the decay. The FDA gave it a "breakthrough therapy designation" two years later.

As a non-invasive and fairly cheap method (it costs about $20-$25 per tooth), SDF treatment, which must be prescribed by a dentist, can save people a lot of money. About 91% of American adults have dental decay, and about 27% have untreated tooth decay, according to the CDC. Tooth decay is common among kids as well -- it's the most common chronic disease in children between 6 and 11 years of age.

3D printing of human organs

Year: 2015

Category: Technology

3D printing technology has improved considerably over the past few years. (Today, low-budget 3D printers are available for anyone who can spare $100.) The technology has advanced so much that producing fully functional replacement organs from a person's own cells seems like a not-so-distant possibility. Scientists at Harvard's Wyss Institute have grown a heart tissue that beats just like a normal human heart.

Production for treatment is still years away, however. The technique, called sacrificial writing into functional tissue (SWIFT), has not even been tested on mice yet. But if it works, it can be used to print other organs, too, potentially saving the lives of thousands of people who are waiting for an organ transplant.

Immunotherapy and cancer

Year: 2016

Category: Treatment

Cancer immunotherapy was named the 2016 Advance of the Year by the American Society of Clinical Oncology. The therapy is designed to support and boost the immune systems response to cancer cells, rather than targeting the cancer itself. One of the most successful immunotherapies so far is the checkpoint inhibition. It makes the immune response stronger by keeping immune cells activated, which does not normally happen when a person has cancer.

It may take decades until immunotherapy could replace the current standards in cancer treatment of surgery, chemotherapy, and radiation, but currently hundreds of immunotherapy drugs are being tested in clinical trials on people.

Some benefits of immunotherapy include fewer side effects than radiation or chemotherapy, lower risk of relapse, and making other cancer treatments more effective.

Opioid crisis recognized as national public health emergency

Year: 2017

Category: Public health

Every day over 130 people in the United States die from opioid overdose, including pain medication, heroin, and synthetic opioids such as fentanyl, according to the National Institutes of Health. In 2017, President Donald Trump declared the opioid crisis a national public health emergency, giving hope that the federal government's involvement could help fight the worst drug crisis in U.S. history.

The official designation removed certain administrative requirements for accessing federal funds to fight the epidemic, including the use of taxpayers' money to make addiction treatments and naloxone, a life-saving medication that can reverse an opioid overdose, drug, more accessible.

The Department of Health and Human Services has renewed the opioid crisis' status as a national emergency several times since 2017. Money has been used to speed up a survey on whether and how often doctors prescribe opioids and help launch anti-addiction programs quicker, according to the a 2018 report by the Government Accountability Office.

Early-stage Alzheimer's treatment

Year: 2019

Category: Diseases

Currently, there is no treatment for Alzheimer's disease, the sixth leading cause of death in the United States. Pharmaceutical companies and universities have tried to tackle different aspects of the neurodegenerative disorder, but to no avail. Until just a few months ago.

Biogen, a biotechnology company, announced in October 2019 it would ask the FDA to approve its Aducanumab drug as first treatment for early Alzheimer's disease. The company said that patients in the early stages of the disease who were treated with a high dose of the drug experienced significant improvements in memory, orientation, and language. If Aducanumab is approved, it will be one of a handful of drugs approved to treat the disease.

Smoking rates at all-time low

Year: 2018

Category: Habits

The short and long-term health problems smoking causes have been well-documented for decades. Today cigarette smoking among U.S. adults is at an all-time low -- 13.7% in 2018, according to the CDC.

While smoking regular cigarettes is down, smoking e-cigarettes is on the rise. About 37% of 12th graders reported vaping in 2018, compared with 28% in 2017. A recent Gallup survey found that 20% of 18- to 29-year-olds vape regularly, more than twice the national average for all age groups.

There has been a recent outbreak of lung injury associated with the use of e-cigarettes. At least 47 deaths and 2,290 lung injuries have been confirmed by the CDC as a result of vaping as of Nov. 20, 2019. The agency has identified vitamin E acetate, an additive in some THC-containing e-cigarettes, as the likely cause for the lung injuries.

Cystic fibrosis treatment approved by FDA

Year: 2019

Category: Treatment

About 30,000 Americans live with cystic fibrosis, a fairly common genetic disease that affects the lungs and other organs, limiting one's ability to breathe as the disease progresses. About 1,000 new cases are diagnosed every year.

The FDA approved in 2019 what it called a "new breakthrough" therapy to treat the condition. The medication, sold under the name Trikafta, is available to patients who are 12 years or older and have the F508del mutation, the most common cystic fibrosis mutation. It is found in 90% of the people living with the disease. The treatment can increase the life expectancy of patients, which is now around 44 years.

Second HIV patient goes into remission

Year: 2019

Category: Diseases

A second person since HIV was identified in the 1980s has been said to be in sustained remission. The patient, who was treated in London, has not been given antiretroviral therapy for 18 months, and the virus has remained undetectable. The good news comes more than a decade after the Berlin patient, known as the first person to have been cured from the infection. Both patients received a stem cell transplant.

HIV, the virus that causes AIDS, is one of the most serious global health challenges. Almost 38 million people live with HIV worldwide, according to the World Health Organization. Just over 60% are receiving treatment.

Blood test detects breast cancer 5 years early

Year: 2019

Category: Diagnoses

Even though deaths from breast cancer have declined, the disease remains the second leading cause of cancer death among women in the United States, according to the CDC. More than 40,000 women die from it a year.

Improved rates of early detection have helped drive up survival rates. A recent British study offers hope that the condition could now be detected five years before there are any clinical signs of it. The new method is a blood test that identifies the body's immune response to antigens produced by tumor cells. The test may be available in clinics in about five years.

Finding a cure for arthritis

Year: 2019

Category: Treatment

2019 has been an exciting year in the field of health technology and scientific research. In addition to such technological developments as organ printing and gene editing, recent research has shown promise for a cure for arthritis. Millions of people suffering from joint inflammation -- from osteoarthritis, for example, which is the most common form of arthritis -- may be helped.

A recent study published in the Science Advances journal has found that "cartilage in human joints can repair itself [...] to regenerate limbs." The body was previously believed to be unable to do so. People have a molecule that helps with joint tissue repair, and that molecule is more active in ankles and less active in knees and hips. The findings can help develop treatments that may prevent, slow, or even reverse arthritis.

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US Network of Specialized Angelman Clinics to Double Thanks to New Partnership – Angelman Syndrome News

As part of her medical training, North Carolina pediatrician Elizabeth Jalazo had studied a little about Angelman syndrome.

I knew enough about it to answer a board question, but I didnt know the complexity of the disorder, she said. Im sure most general pediatricians would share the same sentiment.

Then Jalazo gave birth to a daughter with Angelman and she quickly became an expert on the disease. Evelyn, now 5, is the second of three siblings; theres also Anna, 7, and Charlie, 2.

I didnt know immediately that she had Angelman, but I knew there was something different about Evelyn within five days of her being born, Jalazo said. As most families, we endured a yearlong odyssey of diagnosis. And that was only because I was a clinician at a hospital that had a developmental center where I could push for her to be evaluated.

At the time, Jalazo was a chief resident at Baltimores Kennedy Krieger Institute a division of Johns Hopkins University so I could pick up the phone and ask a professional favor. I basically skipped 9-12 months of wait time.

Jalazo now is a full-time clinician at the University of North Carolina (UNC) School of Medicine in Chapel Hill, where she sees kids with Angelman, Hunter syndrome, and other genetic disorders.

For the past six months, shes also been director of clinical integration at the Chicago-based Angelman Syndrome Foundation (ASF), a nonprofit group dedicated to finding a cure for the severe neurological disorder that affects Evelyn and about 25,000 other Americans.

The U.S. network of specialized clinics Jalazo oversees is now set to more than double in size from eight to 18 thanks to a new partnership between the ASF and the Dup15q Alliance. The network also includes four non-U.S. clinics, in Argentina, Canada, Israel, and the Netherlands.

The 15q Clinical Research Network, as the joint effort is known, will serve patients with Angelman and dup15q syndromes both rare conditions that stem from mutations in the same region of chromosome 15.

In Angelman, the mutation is most commonly a microdeletion on chromosome 15 encompassing theUBE3A gene. This prevents the production of the UBE3A enzyme in certain brain regions.

Partnering with the Dup15q Alliance will allow us to increase our reach to the AS community and provide the best care for our families, Amanda Moore,the foundations CEO, said in a press release. Comprehensive and specific care is critical for AS families throughout the stages of their journey by partnering with Dup15q Alliance, we are able to reach thousands more families with care and support by bringing AS clinics to their location.

The ASF operates on an annual budget of $2 million and uses half of that to fund research. It currently serves 3,500 patients in the database and has around 22,000 followers on social media.

The network Jalazo supervises began in 2011, when three specialized Angelman facilities at Massachusetts General Hospital in Boston, UNCs Carolina Institute for Developmental Disabilities in Chapel Hill, and the NYU Langone Medical Center in New York City all became ASF-supported clinics. Six more were added over the last eight years.

Patients travel to where they can receive this evidence-based care, said Jalazo, who spoke to Angelman Syndrome News at the 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit, organized by the National Organization for Rare Disorders.

When its challenging to travel with a child that has special needs especially one with a rare disorder that no ones ever heard of it becomes incredibly beneficial to see all the specialists you need to see in one or two days, she said.

Most of these clinics operate one day a month, though some sites such as Mass General see patients weekly. The current U.S. network treats around 700 Angelman patients throughout the United States; including the four foreign clinics, the ASF network serves close to 1,000 patients.

In December 2018, Israels Sheba Medical Center at Tel HaShomer joined the network. That specialized AS clinic, housed at Shebas Edmond and Lily Safra Childrens Hospital just outside Tel Aviv, was established seven years ago by pediatric neurologist Gali Heimer. Since then, its evolved into the fourth-largest specialized Angelman syndrome clinic in the world, treating 85 of Israels known 100 or so Angelman patients.

Thanks to the new agreement between ASF and Dup15q Alliance both based in the Chicago area 10 Angelman clinics will join the ASF network, including Seattle Childrens Hospital, MiamisNicklaus Childrens Hospital, and the Intermountain Primary Childrens Medical Center in Salt Lake City, Utah.

This isnt the first time ASF and Dup15q Alliance have worked together. Because the two syndromes are similar, the groups have co-hosted a research symposium every two years. Many researchers who study Angelman also study dup15q.

Our goal is to bring together all the providers caring for Angelman individuals across the globe, Jalazo said. We encourage our sites to be multidisciplinary, so that patients can receive comprehensive care, but we do recognize that some clinicians around the world are not seeing patients in a multidisciplinary setting.

As clinical director at the ASF, Jalazo oversees a monthly virtual meeting via Zoom where representatives of all the clinics talk about pressing issues.

One of the biggest challenges is harnessing the power of patient data, she said. Every time a patient shows up at a clinic, an incredible amount of data is collected thats valuable to patient care, to understanding the natural history of the disease, and to drug development. So we want to harness that and ensure were utilizing and formatting it in a way thats accessible.

Jalazo said that when it comes to her own daughter, an early diagnosis definitely helped. Evelyn has been on a modified ketogenic (low-carb, high-fat) diet from a very early age.

She eats whatevers put in front of her, said the pediatrician, adding that Evelyn generally eats eggs and avocados every morning. Shell go find the blender and if its 3 p.m. and I havent made a smoothie, shell bang the cup on the counter.

Indeed, though the little girl cant speak, she does communicate.

If she needs something, shell go get it and bring it to me. Shell force eye contact. Before she could even walk, shed scoot over to you and direct your gaze over to whatever she wanted you to see, Jalazo said. I think she understands most of what I tell her. There are certain tones she uses. Her term of endearment is a g sound which means I love you.

Evelyns sister, Anna, is a natural caretaker and knows intuitively what Evelyn needs, while Evelyn and Charlie have a very typical sibling relationship they fight all the time, Jalazo said.

Shes completely nonverbal and youd assume she cant read the cues of social situations, but she and Charlie communicate 100% effectively, she said.

As for a cure for AS, Jalazo said gene therapy is extremely promising but that short-term, the focus is on antisense oligonucleotides, or ASOs. These are small pieces of DNA or RNA that can bind to specific molecules of RNA and reduce, restore, or modify protein expression.

Its an incredibly exciting time for the Angelman community, in terms of promising therapeutics coming down the pipeline, one of which is gene therapy, she said. In the immediate future, well have the opportunity to see the impact of non-gene therapy approaches like ASOs. This will give us a little sneak peek into what gene therapy can do.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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US Network of Specialized Angelman Clinics to Double Thanks to New Partnership - Angelman Syndrome News

ICMR issues guidelines for gene therapy in India – Daily Pioneer

To ensure that CRISPR gene editing technology is not misused as was done by a Chinese scientist who created the gene-edited babies recently that attracted global criticism, the Indian Council of Medical Research (ICMR) has framed national guidelines and regulations regarding the procedures and requirements to be followed for performing Gene Therapy in India.

CRISPR stands for clustered regularly interspaced short palindromic repeats are DNA sequences found within the genome of prokaryotes, and are used in genome editing along with enzymes called CRISPR-associated nucleases (most commonly Cas9).

Gene Therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

Since this nascent field is emerging in India, the Government has proactively have come up with the National Guidelines for Gene Therapy Product Development and Clinical Trials to promote further research and streamline regulatory processes for future clinical trials using gene therapeutic products (GTPs).

As per the New Drugs and Clinical trial Rules (2019) the GTPs falls under new drug and shall always be deemed to be new drug. Thus as per these rules framed jointly by Indian Council of medical Research and Department of Biotechnology (DBT), academic trials are not applicable to clinical trials using GTPs.

"India has large burden of genetic disorders and unmet medical needs and gene therapy can prove to be a turning point in treatment of such disorders. However, it also brings along with it unique technical risks and ethical challenges," said an official from the ICMR.

She cited the creation of babies using germline gene editing by a Chinese scientist recently that has fuelled a debate on ethical concerns regarding applications of gene therapy technologies.

"This also brought to forefront the requirement of stringent guidelines and regulations to prevent misuse and premature commercialization.

"Many countries around the world have developed rules and guidelines to regulate gene therapy trials.

Taking cognizance of situation, it was felt necessary to frame national guidelines and regulations to direct scientists and clinicians including industry regarding the procedures and requirements to be followed for performing gene therapy in India," she added.

It is proposed to establish Gene Therapy Advisory and Evaluation Committee (GTAEC) anchored at ICMR. GTAEC shall be an independent body of experts representing diverse areas of biomedical research, concerned government agencies and other stakeholders.

"This committee will be composed of a core group of scientists and clinicians in the sector, as well as representation of the government agencies (ICMR, DGHS, CDSCO, DBT, DST, MCI). For each disease area in GTP trials, specific clinical consultants with extensive disease specific expertise will be co-opted to aid in the decision-making process," as per the guidelines.

It says that biological material from humans can be procured only from clinics/hospitals that have an evaluation committee.

The EC must ensure that the Standard Operating Procedures (SOPs) are in compliance with the national guidelines. "Investigators should treat the biological material with utmost respect and adequate care to avoid its misuse. The institute needs to define SOPs for development, production; storage and disposal of the GTPs or its components should be as per the Regulations and Guidelines on Bio-safety of Recombinant DNA Research and Bio-containment 2017," says the guidelines.

Until 2017, almost 2600 gene therapy and 6 clinical trials have been conducted worldwide in 38 countries, of which 64.9 per cent were in USA, 23.2 per cent in Europe and approximately 6.5 per cent were in Asia.

Within Asia, China has reported about 84 gene therapy clinical trials, followed by Japan (44 trials) and South Korea (14 trials). The vast majority of gene therapy trials have addressed cancer (66.6 per cent), monogenic diseases (11.5 per cent), cardiovascular diseases (6.2 per cent) and infectious diseases (6.3 per cent)

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Mergers and Acquisitions Will Strengthen Payer Identity in 2020 – HealthPayerIntelligence.com

December 11, 2019 -Payers will seek to strengthen their identities in 2020 and they will use mergers and acquisitions as the tools for transformation, according to a recent HRIs PricewaterhouseCoopers (PwC) report.

In 2020, organizations will make strategic deals not to just grow larger but instead to expand into new identities with platforms anchored in value, innovation, customer experience and population health, the report stated. As they weigh their options, health companies will need to ensure that the deals they pursue pass the sniff test of employers and consumers seeking more affordable care.

Forty percent of healthcare executives from across the industryincluding payers, providers, pharmaceutical companies, and othersare somewhat or very likely to work with or acquire companies from other sectors of the industry in the new year. When making these deals, 26 percent of payer executives prioritize finding partners that maintain or increase their companies competitive advantages.

The report highlights four identities that payers and other healthcare organizations can embrace in 2020 that may influence their deal-making decisions.

Some payers may choose to emphasize their strengths as product and innovation leaders. To achieve this, they will seek partnerships with providers and researchers who have acclaimed specialty skillsets. These payers may also focus on acquiring healthcare technology brands to support the payers technological superiority and advancements.

In step with this prediction, a KLAS report from earlier in 2019 revealed that payers are moving away from systems that they have relied on for a long time. Instead, they seem to favor partnering with new, disruptive technologies. These partnerships can help develop an innovative public image.

Cignas deal with Express Scripts is an excellent example of a payer making a deal to enhance its identity as a technology leader. Because of that partnership, the payer has been able to develop a new gene therapy payment model with Embarc.

Payers that strive to be population health and outcomes leaders will require strong relationships and excellent communication with provider and advocacy groups. These payers should also have an excellent grasp of community health and access to care challenges.

Companies choosing this identity may look to acquire smaller organizations focused on key patient segments as they focus product portfolios and develop patient engagement services to ensure optimal outcomes, PwC suggested.

PwC pointed to Clovers Medicare Advantage plan and its Clover Therapeutics medicinal research branch as an example of a successful leader in population health management and patient outcomes.

Other payer may seek to become member experience and consumer advocacy leaders in 2020. In this case, finding partners that can improve access to care, ease, and healthcare payer services are key to elevating the patient experience.

Partnerships in this category are aiming for longevity with their members, with the goal of retaining members for their lifetimes. Facilitating worksite clinics can be one way to improve the member experience by making access to healthcare highly convenient.

Value leadership was responsible for some of the biggest deals of 2019, according to PwC, and it will continue to be a major driver in 2020. For example, Optum acquired DaVita Medical Group and CVS Health Hartford likely because they wanted to enhance their value leadership identity, PwC suggested.

However, as important as these categories may be, the report urged payers and healthcare companies to think outside of the box and potentially combine different identities to make a new one.

The product leader in the future will be a much harder sell, King Li, MD, dean of the Carle Illinois College of Medicine and chief academic officer of the Carle Health System predicted in the report. Its hard to say you are a product leader when people make decisions based on the same AI support. In this case, patient experience will become much more important in achieving a positive outcome.

One trend that payers considering a merger or acquisition next year will need to keep in mind is the patient and employer perspective. These demographics are the most affected by payer mergers and are largely ambiguous on their sentiments toward healthcare mergers. Payers need to keep patient and employer communication lines open to inform them of changes in their coverage during mergers and listen to patients and employers needs.

Lastly, PwC proposed that payers and healthcare organizations looking to make deals and improve their identities find a private equity partner.

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University of Iowa researchers say $18 million grant could soon lead to cure for rare brain disease – KCRG

IOWA CITY, Iowa (KCRG) - Researchers at the University of Iowa Hospitals and Clinics received an $18 million federal grant to continue research on a rare brain disorder. They say that could lead them to ultimately finding a cure.

Dr. Peg Nopoulos, a neuroscientist with UIHC says they have been studying Huntington's disease on children that are at risk of acquiring the disease. The fatal brain disease is genetic, currently has no cure, and can affect how a person acts or thinks.

Nopoulos said the grant funding that they will receive over the next five years from the National Institutes of Health will help them complete their clinical trial for the disease, that is currently in the third phase. She said that could lead to positive results to combat the disease.

"We're looking to see if that therapy can slow progression," Nopoulos said. "The ultimate goal is if those trials are successful, then we're going to want to give it to people who are at-risk and who carry the gene but have not yet had the disease. We want to prevent it."

Nopoulos said she believes based on their research, a cure for Huntington's disease could come sooner rather than later.

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University of Iowa researchers say $18 million grant could soon lead to cure for rare brain disease - KCRG