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FDA issues draft guidance on developing drugs for Duchenne Muscular Dystrophy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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Sold Out 2015 Bank of America Chicago Marathon and Marine Corps Marathon Entries Still Available through MDA Team Momentum

National Endurance Program Actively Recruiting Participants for Signature Half / Full Marathon Events this Fall CHICAGO, June 1, 2015 — The Muscular Dystrophy Association is offering an exclusive opportunity for anyone interested in participating in the sold out 2015 Bank of America Chicago and Marine Corps Marathons to secure guaranteed entry by signing up through . . .

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MDA Summer Camp Celebrates 60 Years of Changing Lives

Barrier-free, weeklong camp for kids with neuromuscular disease offers ‘Awesome Adventures’ CHICAGO, May 20, 2015 – The Muscular Dystrophy Association (MDA) is set to kick off another season of summer camp. Now in its 60th year, MDA summer camp supports families by hosting thousands of kids fighting muscular dystrophy and other life-threatening diseases that severely . . .

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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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BioMarin Completes Rolling NDA Submission for Treatment of Duchenne Muscular Dystrophy

Press Release: BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “This is very encouraging news for the DMD community, and we hope it means that a treatment is on the horizon. If drisapersen were to . . .

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Western Refining and MDA Join Forces to Help Send Kids with Muscle Disease to Summer Camp

Stores across the Southwest to raise vital funds through MDA’s summer camp mobile program PHOENIX, April 28, 2015 — More than 260 Western Refining convenience stores, including Giant, Mustang, Sun Dial and Howdy’s, throughout the Southwest will raise money to help the Muscular Dystrophy Association save and improve the lives of kids fighting muscular dystrophy . . .

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