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Archives
Category Archives: Muscular Dystrophy Treatment
PTC Therapeutics Announces First Publication of Real-World Data Showing Translarna (ataluren) Significantly Preserves Ability to Walk for Longer in…
Posted: Published on February 5th, 2020
SOUTH PLAINFIELD, N.J., Feb. 3, 2020 /PRNewswire/ --PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Continue reading
Posted in Muscular Dystrophy Treatment
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Global Gene Therapy Market is Projected to Reach USD 13.0 Billion by 2024 from USD 3.8 Billion in 2019, at a CAGR of 27.8% – ResearchAndMarkets.com -…
Posted: Published on February 5th, 2020
DUBLIN--(BUSINESS WIRE)--Feb 5, 2020-- The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering. High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. Continue reading
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Catabasis Pharmaceuticals Announces Closing of Public Offering of Common Stock and Full Exercise of Over-Allotment Option – Yahoo Finance
Posted: Published on February 5th, 2020
Catabasis Pharmaceuticals, Inc. Continue reading
Posted in Muscular Dystrophy Treatment
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Global Gene Therapy Industry Outlook 2020-2024 – Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities – Yahoo Finance
Posted: Published on February 5th, 2020
DUBLIN, Jan. 30, 2020 /PRNewswire/ -- The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering Continue reading
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French cancer-focused microbiome player is flush with 18M Series B injection – Endpoints News
Posted: Published on February 5th, 2020
Last year, the death of an immuno-compromised elderly patient in a fecal microbiota transplantation trial due to a donation that contained a rare type of E. coli bacteria sent shivers across the field. The incident marred an otherwise exploding field of drug development that backed replenishing the gut with good bacteria as a safe and effective means to fortify the immune system to fight disease. Continue reading
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Duchenne Muscular Dystrophy: Muscle Weakness That Begins In This Body Part Could Be A Warning Sign – International Business Times
Posted: Published on February 3rd, 2020
KEY POINTS Duchenne muscular dystrophy (DMD) is a rare muscle disorder affecting about 1 in 3,500 male births globally. The condition is usually recognized between the ages of three and six. Continue reading
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Third Time’s the Charm as Acceleron Drug Shows Early Promise in PAH – Xconomy
Posted: Published on February 3rd, 2020
Xconomy Boston Sotatercept was one of the first compounds to emerge from the labs of Acceleron Pharma, but it became a forgotten piece of the companys pipeline. Continue reading
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Six patients with rare blood disease are doing well after gene therapy clinical trial – UCLA Newsroom
Posted: Published on February 3rd, 2020
Sarah C.P. Continue reading
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Did FDA lower the bar in its Sarepta decisions? We discuss – STAT
Posted: Published on January 27th, 2020
Containing new coronavirus may not be feasible, experts say, Containing new coronavirus may not be feasible, experts say, as they warn of possible sustained global DNA sleuths read the coronavirus genome, tracing its origins DNA sleuths read the coronavirus genome, tracing its origins and looking for dangerous mutations How fast can biotech come up with a vaccine How fast can biotech come up with a vaccine for the latest outbreak? Continue reading
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Is the Medication You’re Taking Worth Its Price? – Undark Magazine
Posted: Published on January 27th, 2020
Austin was three years old and Max was a newborn when their mother, Jenn McNary, learned they had a rare genetic condition called Duchenne muscular dystrophy. Continue reading
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