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
  • By Karen Harley
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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
  • By Karen Harley
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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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24 Mar
  • By Karen Harley
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ReveraGen BioPharma Receives FDA Fast Track Designation for Vamorolone for the Treatment of Duchenne Muscular Dystrophy

ROCKVILLE, Maryland, March 24, 2017 ReveraGen BioPharma Inc, a privately held corporation, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for vamorolone (VBP15) for the treatment of patients with Duchenne muscular dystrophy.  This designation can speed the review of efficacy and safety data for vamorolone in boys with […]

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http://marathonpharma.com/news/2017/02/emflaza-deflazacort-just-fda-approved/
FDA Approves EMFLAZA™ (deflazacort) Tablets and Oral Suspension for the Treatment of Duchenne Muscular Dystrophy in Patients 5 Years and Older
February 9, 2017 leilanifera

Marathon Pharmaceuticals, LLC (Marathon), a U.S. research-based biopharmaceutical company focused solely on the development of new treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted approval of EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy in patients 5 years and older. Duchenne, a severe form of muscular dystrophy, is a rare disease and fatal genetic disorder that affects about 15,000 people in the United States.1

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http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm
FDA approves drug to treat Duchenne muscular dystrophy
February 9, 2017 leilanifera

The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.

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