15 Nov
  • By Karen Harley
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Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor’s Investigational Cell Therapy

ANAHEIM, Calif., Nov. 15, 2017 /PRNewswire/ — Boys and young men in advanced stages of Duchenne muscular dystrophy experienced significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function, following treatment with CAP-1002, the lead investigational therapy under development at Capricor Therapeutics (NASDAQ: CAPR). These findings were reported today by […]

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08 Nov
  • By Karen Harley
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Exonics Therapeutics Raises $40 Million in Series A Financing

Funds to support continued development of SingleCut CRISPR technology for Duchenne muscular dystrophy CAMBRIDGE, Mass. – November 8, 2017 – Exonics Therapeutics, Inc., a biotechnology company focused on developing SingleCut CRISPR technology to repair mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, today announced it has secured a $40 million Series A financing to advance […]

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04 Oct
  • By Karen Harley
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Capricor Therapeutics Presents Positive Six-Month Results in Duchenne Muscular Dystrophy at World Muscle Society International Congress

SAINT MALO, France, Oct. 4, 2017 /PRNewswire/ — Capricor Therapeutics, Inc. (NASDAQ: CAPR), in its presentation today at the 22nd Annual International Congress of the World Muscle Society, reported that teens and young men in the advanced stages of Duchenne muscular dystrophy (DMD) experienced meaningful improvements in cardiac and upper limb function after a single […]

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04 Oct
  • By Karen Harley
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Catabasis Pharmaceuticals Reports Positive Results from Open-Label Extension of Phase 2 MoveDMD® Trial Evaluating Edasalonexent in Duchenne Muscular Dystrophy and Plans to Initiate Phase 3 Clinical Trial in First Half 2018

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 4, 2017– Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new positive efficacy results showing sustained disease-modifying effects in the MoveDMD trial open-label extension following 24 and 36 weeks of treatment with edasalonexent. Across all key assessments of muscle function, improvements were observed in the rate of decline after 24 […]

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27 Jul
  • By Karen Harley
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Sarepta Therapeutics Announces its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data in Nature Communications

Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today […]

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22 Jun
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Santhera’s Raxone® Receives First Positive EAMS Scientific Opinion from UK’s MHRA in Duchenne Muscular Dystrophy

Liestal, Switzerland, June 22, 2017 – Santhera Pharmaceuticals (SIX: SANN) announces that the UK`s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for patients with respiratory function decline not taking glucocorticoids in Duchenne Muscular Dystrophy (DMD). The aim of the EAMS is […]

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22 Jun
  • By Karen Harley
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Sarepta Therapeutics and Genethon Announce a Gene Therapy Research Collaboration for the Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass. and EVRY, France, June 21, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, a non-profit R&D organization dedicated to the development of biotherapies for orphan genetic diseases from research […]

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25 Apr
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Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy

LOS ANGELES, April 25, 2017 /PRNewswire/ — Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions, today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an […]

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20 Apr
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PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne Muscular Dystrophy in the U.S.

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it has completed its acquisition of all rights to Emflaza™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017. “We are pleased the acquisition was completed […]

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19 Apr
  • By Karen Harley
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CRISPR Corrects Duchenne-Causing Mutations

Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model. By Anna Azvolinsky | April 12, 2017 http://www.the-scientist.com/?articles.view/articleNo/49190/title/CRISPR-Corrects-Duchenne-Causing-Mutations/ Researchers have been studying the CRISPR-Cas9 gene-editing system as a potential therapeutic tool to modify or delete pathogenic sequences within the human genome. Now, […]

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