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 . . .
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 . . .