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