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 . . .
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 . . .
By Amanda M. Haidet-Phillips, Ph.D., MDA’s ALS Scientific Program Officer Throughout 2014, everyone from celebrities to school children to MDA’s own CEO could be seen dumping ice water over their heads to raise awareness and dollars to fight ALS. It was a phenomenon that shined an urgently-needed spotlight on a disease that steals everyday freedoms . . .
Stores Nationwide Raise More Than $7.5 Million during 2015 Shamrock Program to Help Improve Lives in MDA Communities #MDAshamrocks MDA’s 2015 National Goodwill Ambassador Reagan Imhoff, along with her parents Jenny and Joe, helped spread awareness to Lowe’s employees and customers about the importance of the MDA shamrock and how funds raised through the program . . .
Press Release: Santhera’s Positive Phase III Trial (DELOS) in Patients with Duchenne Muscular Dystrophy Published in The Lancet “This is welcome news for our community,” said MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D. “The need for effective DMD therapies is urgent, and we look forward to the possibility that idebenone may . . .
Press Release: Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystrophy Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “This is exciting news for our community. MDA is committed to bringing safe and effective treatments and cures to kids and adults living with neuromuscular . . .
Press Release: BioBlast Pharma Announces Granting of Fast Track Designation by the FDA for Cabaletta in Oculopharyngeal Muscular Dystrophy (OPMD) Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “We are thrilled to see this continued progress from the FDA. OPMD is a rare and debilitating muscular dystrophy for which there . . .
Marshall Tucker Band to headline exclusive concert at 28th annual event #MDARide4Life PHILADELPHIA, April 7, 2015 — Hundreds of motorcycle enthusiasts participating in MDA Ride for Life this spring will put their kickstands down in an all new location for the 28th annual event to benefit the Muscular Dystrophy Association on May 1- 3 at . . .
MDA, Genzyme and Emory University team up to expand access to genetic testing for accurate diagnosis of limb-girdle muscular dystrophy CHICAGO, March 26, 2015 — The Muscular Dystrophy Association announced today that thanks to a grant from Genzyme, a Sanofi company, and in collaboration with Emory Genetics Laboratory, MDA clinics are now offering genetic testing . . .
Saturday morning’s presentations were devoted to the latest news about clinical trials and drug development. Among the highlights: Duchenne muscular dystrophy (DMD): Edward Kaye from Sarepta Therapeutics presented his company’s most recent data about its experimental drug eteplirsen, designed to treat about 13 percent of the Duchenne muscular dystrophy (DMD) population, and discussed plans for . . .