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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
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, . . .
By Jenny Imhoff Today marks the beginning of a long weekend for many Americans. It’s a time to soak up the last days of summer sun and gather with friends and family. For many Americans, Labor Day weekend also has been synonymous with MDA, thanks to its long-running telethon. My family was very lucky to . . .
MDA is committed to accelerating treatments and cures, using innovative ways to engage new supporters CHICAGO, May 1, 2015 — MDA, the Muscular Dystrophy Association, today announced that the new realities of television viewing and philanthropic giving have made this the right time for the organization to move beyond its historic Labor Day telethon. It . . .
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 . . .