Articles

Santhera Updates on U.S. Regulatory Filing for Raxone ® (idebenone) in Duchenne Muscular Dystrophy (DMD)

July 14, 2016

Santhera Pharmaceuticals (SIX: SANN) announces that it has received written correspondence from the U.S. Food and Drug Administration (FDA) on its proposed subpart H approval pathway for Raxone in DMD patients not taking concomitant glucocorticoids.

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Catabasis Pharmaceuticals Announces the Initiation of an Open- Label Extension for the MoveDMD ® Trial Studying Edasalonexent (CAT -1004) in Duchenne Muscular Dystrophy

July 7, 2016

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of an open- label extension for the Phase 2 portion (Part B) of the MoveDMD trial studying edasalonexent (CAT-1004), an investigational therapy, in boys with Duchenne muscular dystrophy (DMD). The Phase 2 portion of the MoveDMD trial is a randomized, double-blind, placebo-controlled 12-week trial to assess the efficacy and safety of two doses of oral edasalonexent with the primary end point being change in magnetic resonance imaging (MRI).

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NHS England Enables Access to Translarna™ ▼ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy

July 7, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company and NHS England have successfully negotiated a Managed Access Agreement (MAA) for Translarna (ataluren) for ambulatory patients aged five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD). This decision provides reimbursed patient access to Translarna in England via a five-year MAA. Translarna previously received a positive recommendation from the National Institute for Health and Care Excellence (NICE) in April of 2016, subject to PTC and NHS England finalizing the terms of the MAA. NICE is expected to issue final guidance later this month following execution of the MAA, with implementation soon after.

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Capricor Therapeutics Provides Enrollment Update on HOPE Clinical Trial in Duchenne Muscular Dystrophy

June 23, 2016

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today announced that patient enrollment in its ongoing randomized HOPE-Duchenne clinical trial (Halt cardiomyOPathy progrEssion in Duchenne) has exceeded 50% of its 24-patient target.

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Pluristem Reports Data Showing PLX -PAD Cells Effective in Treating Duchenne Muscular Dystrophy

June 22, 2016

Pluristem Therapeutics Inc . (NasdaqCM: PSTI, TASE: PSTI/ PLTR), a leading developer of placenta- based cell therapy products, today reported positive data from preclinical studies of its PLX -PAD cells in the treatment of Duchenne muscular dystrophy.

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