Sarepta Completes NDA for Eteplirsen and FDA Accepts BioMarin NDA for Drisapersen; Two DMD Treatments on Horizon

Background: On Monday, June 29, BioMarin announced that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.  Both “exon-skipping” drugs target exon 51, and may help up to 13% of Duchenne muscular dystrophy patients. Exon skipping is a treatment strategy in which sections of genetic . . .

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MDA Affirms Commitment to Safe Therapies

Statement from Kristin Stephenson, Vice President Policy & Advocacy Muscular Dystrophy Association:The Muscular Dystrophy Association (MDA) is committed to bringing safe and effective treatments and cures to children and adults living with life-threatening neuromuscular diseases as quickly as possible.  To this end, MDA has invested more than $1 billion in research grants that fund groundbreaking . . .

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Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Type 2 and 3 SMA

Press Release: Continued increases observed in multiple measures of muscle function in SMA children treated with ISIS-SMN Rx Statement from MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D.: “We are excited to see that Isis Pharmaceuticals reported positive results from patients who were enrolled in their Phase 2 trial and who continued to receive the drug after the . . .

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Akashi Therapeutics Reports Data from Phase 1b-2a Study in Boys with Duchenne Muscular Dystrophy

Press Release: Akashi Therapeutics Reports Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “Data from this ongoing clinical trial shows that boys with DMD who were treated with HT-100 demonstrated improvements in muscle strength. If these positive trial results are borne out in future . . .

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News: Isis Pharmaceuticals Reports Data from Phase 2 Study in Infants with Spinal Muscular Atrophy

On June 11, Isis Pharmaceuticals, Inc. released an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA).  The data showed that infants treated with ISIS-SMNRx have shown continued increases in median event-free survival, positive results in muscle function tests, and attainment of important developmental . . .

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FDA issues draft guidance on developing drugs for Duchenne Muscular Dystrophy

Background:  On June 9th, the U.S. Food and Drug Administration (FDA) released a draft containing guidance to assist drug companies working to develop drugs for the treatment of Duchenne muscular dystrophy (DMD) and related diseases including Becker muscular dystrophy, DMD-associated cardiomyopathy and symptomatic DMD carrier status in females. The guidance relates FDA perspective on a number . . .

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FDA Approves Investigational New Drug Application for ALS Cellular Therapy

Press Release:  Novel Cellular Therapeutic Approach for ALS Gets FDA Clearance for First-In-Human Trials Statement from MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D.: “This is a promising development in the quest for treatments and cures for ALS. Approval of the Investigational New Drug Application (IND) by the FDA clears the way for a clinical trial of . . .

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28th Annual MDA Ride for Life Raises More Than $1.1 Million to Fuel the Fight Against Muscle Disease

PHILADELPHA, May 11, 2015 — Opening weekend at Camelback Lodge and Indoor Waterpark helped bring huge success to the 28th annual MDA Ride for Life event held on May 1-3, which raised more than $1.1 million to benefit families affected by muscular dystrophy and related life-threatening diseases. Located in the heart of the Pocono Mountains, . . .

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MDA is committed to accelerating treatments and cures, using innovative ways to engage new supporters CHICAGO, May 1, 2015 — MDA, the Muscular Dystrophy Association, today announced that the new realities of television viewing and philanthropic giving have made this the right time for the organization to move beyond its historic Labor Day telethon. It . . .

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