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 […]
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 […]
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, […]
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 […]
Exonics to develop a therapeutic approach to correct underlying genetic mutations in Duchenne, a degenerative genetic disorder without effective treatment options to halt progression of disease.

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

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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

FDA approves drug to treat Duchenne muscular dystrophy

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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.

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

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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).

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

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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.

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

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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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