MDA Awards $10 million in new Research Grants

Record-breaking interest highlights progress, desire to find breakthroughs across diseases CHICAGO, August 25, 2015 – Powered by its big-picture perspective to accelerate treatments and cures across the broad spectrum of neuromuscular diseases, MDA today announced the award of $10 million in new research grants to the world’s brightest scientists conducting leading-edge discovery for muscular dystrophy, ALS and . . .

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FDA Accepts Sarepta NDA for Eteplirsen to Treat DMD

Background: Sarepta announced today that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for eteplirsen to treat Duchenne muscular dystrophy (DMD). Eteplirsen is an “exon-skipping” drug that targets a section of DNA called exon 51, and may help up to 13 percent of Duchenne muscular dystrophy (DMD) patients. Exon . . .

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Marathon Pharmaceuticals Begins New Drug Application Process for Deflazacort to Treat Duchenne Muscular Dystrophy

Following a pre-NDA meeting with the U.S. Food and Drug Administration (FDA), Marathon Pharmaceuticals has announced that it will begin the New Drug Application (NDA) process for deflazacort as a treatment for Duchenne muscular dystrophy (DMD). Marathon expects to submit the NDA in the first quarter of 2016. If the application is approved, the company . . .

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FDA Approves Drug for Primary Hyperkalemic and Hypokalemic Periodic Paralysis

Statement from MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D.: “We couldn’t be more pleased for our families affected by hyperkalemic and hypokalemic periodic paralysis, as the first treatment for these diseases has been approved by the U.S. Food and Drug Administration (FDA) and is expected to be available in . . .

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Major League Baseball Joins MDA and Fourteen Other ALS Organizations to Launch ALS Ice Bucket Challenge 2015

CHICAGO, July 31, 2015 —The 2015 ALS Ice Bucket Challenge will officially get underway today at Fenway Park in Boston, when Major League Baseball and the Boston Red Sox honor Ice Bucket co-founders Pat Quinn and Pete Frates, the ALS community and Lou Gehrig’s legacy by taking the challenge. MLB is donating $100,000 to further . . .

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MDA and Jiffy Lube Team Up to Support the Fight Against Muscle Disease with Fourth Annual MUSCLE UP!SM Campaign

Relationship with MDA Encourages Drivers to Give Back Through In-Store Donations CHICAGO, July 29, 2015 – The Muscular Dystrophy Association (MDA) is pleased to announce that Jiffy Lube® is hosting its fourth annual MUSCLE UP!SM campaign.  As part of the program, Jiffy Lube invites drivers to visit participating Jiffy Lube® service centers from Aug. 1 . . .

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ALS Ice Bucket Challenge Returning This August

Statement from MDA President and CEO Steven M. Derks: “MDA takes a big-picture perspective to fight ALS — investing in research to find breakthroughs that will lead to treatments and cures, caring for people from day one at our specialized clinics, and helping ALS families in hometowns across America live longer, stronger lives. MDA is . . .

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Western Refining Raises $524,286 to Help Send Kids with Neuromuscular Disease to MDA Summer Camp

Stores across the Southwest exceed 2015 fundraising goal by nearly $100,000. PHOENIX, July 7, 2015 — More than 260 Western Refining convenience stores throughout the Southwest, including Giant, Mustang, Sun Dial and Howdy’s, raised more than $524,286 during the Muscular Dystrophy Association’s summer camp mobile program, surpassing their original 2015 fundraising goal by nearly $100,000, . . .

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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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