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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .