Muscular dystrophy couldn’t keep Paul Robertson from the water, and he doesn’t want muscle disease to stop anyone from doing what they love. “When you get on the water, it’s a sense of freedom. It’s just the thrill of the bite, the thrill of the competition, the thrill of being on the water,” the lifelong . . .
Harvard/MIT scientists and partners are set to spread awareness about neuromuscular diseases in the greater Boston area at the 3rd Annual Strength, Science and Stories of Inspiration (SSSI) 2016. Attend the event, where key stakeholders will come together and facilitate new relationships to accelerate progress toward treatments and cures. In addition, hear about . . .
March 16 marks Jerry Lewis’ 90th birthday. No one has been a greater champion for MDA or MDA families than the legendary comedian, and his heroic efforts have helped millions of Americans living with muscle-debilitating diseases and made possible progress in research that was once thought unimaginable. Will you join us in wishing him well . . .
Chris Lorson, professor of veterinary pathology, and molecular biology and immunology at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies. . . .
Kurt Harzke’s son Jacob was diagnosed with Duchenne Muscular Dystrophy (DMD) when he was three years old. He’s 13 now, a smart, ambitious teen, who suffers from a disease that currently has no cure. Last month, on Rare Disease Day, February 29, Kurt and Jacob spoke to a group from the Muscular Dystrophy Association (MDA) and . . .
The experimental drug eteplirsen, under development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD), is under review by the U.S. Food and Drug Administration (FDA) for market approval. It marks an exciting and historic time for the MDA community and an important milestone for DMD treatments. Before a final decision is rendered, however, an . . .
Marathon Pharmaceuticals announced today that it is expanding patient access to deflazacort, its investigational therapy for Duchenne muscular dystrophy (DMD). Medical centers across the country are now participating in the open-label expanded access program (EAP) called ACCESS DMD to provide deflazacort to qualified U.S. patients at no charge. Deflazacort, a corticosteroid, works as an anti-inflammatory . . .
For Mindy Scheier, the founder of Runway of Dreams, making a difference for the muscular dystrophy community means thinking both long and short term. Since her son Oliver was diagnosed with a rare form of muscular dystrophy, she has channeled her energy into finding a cure. Together with her family, she has helped raise more . . .
In two webcasts held Feb. 29 — one for investors and one for Duchenne muscular dystrophy (DMD) patients and families — PTC Therapeutics addressed the Refuse to File letter it received last week from the U.S. Food and Drug Administration (FDA) stating that PTC’s New Drug Application (NDA) for Translarna (ataluren) to treat DMD was not sufficient . . .
Researchers are looking for people with amyotrophic lateral sclerosis (ALS) to participate in a phase 1 clinical trial, sponsored by Biogen and Ionis Pharmaceuticals, to test the experimental drug BIIB067 (Ionis-SOD1Rx). BIIB067 is an antisense oligonucleotide, composed of short segments of synthetic genetic material (nucleic acid) that bind to RNA. The drug is designed to . . .