Articles

FDA Approves EMFLAZA™ (deflazacort) Tablets and Oral Suspension for the Treatment of Duchenne Muscular Dystrophy in Patients 5 Years and Older

February 9, 2017

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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FDA approves drug to treat Duchenne muscular dystrophy

February 9, 2017

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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Catabasis Pharmaceuticals Announces Top-Line Results for Part B of the MoveDMD® Trial for Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy

January 31, 2017

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced top-line safety and efficacy results for Part B of the MoveDMD® trial of edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD).

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CHMP Recommends Renewal of Translarna's™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment

November 11, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

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Capricor Therapeutics Announces Plans to Expand Clinical Development Program in Duchenne Muscular Dystrophy to Evaluate Peripheral and Respiratory Muscle Function

November 10, 2016

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, today announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications.

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