By Amanda M. Haidet-Phillips, Ph.D., MDA’s ALS Scientific Program Officer As the month of August comes to an end, and the 2015 ALS Ice Bucket Challenge with it, I want to share with you some exciting new research news. MDA has announced the award of 36 new research and development grants, totaling nearly $10 million, . . .
CHICAGO, August, 27, 2015 — Prominent business and community leaders from across the country have been appointed to the Muscular Dystrophy Association’s Board of Directors. As members of the board, these 20 men and women will volunteer assistance and counsel to support MDA’s mission to fund lifesaving research, provide comprehensive care through its services and . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
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, . . .
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 . . .
