Researchers are looking for people with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate.
Amifampridine phosphate is a potassium channel inhibitor designed to cause greater stimulation of muscle by prolonging nerve signals and is expected to help treat muscle weakness in people with CMS. The drug has shown promising results in treating patients with a related disorder called LEMS.
Osaka-based Mitsubishi Tanabe Pharma has reported encouraging 12-month efficacy and safety data for edaravone (brand name Radicava) for the treatment of ALS. Edaravone is delivered by intravenous injection. It’s thought to work by relieving the effects of oxidative stress, which, in people with ALS, has been suspected to play a role in the death of . . .
Few things are better than the perfect gift — except, perhaps, the gift that gives love to the recipient and to a whole community as well. This season, surprise your family with gifts that give to MDA families — and MDA research — too. Good Reads Stack your family bookshelf with must-reads that help explain . . .
OK, friends. I’ve got some really great news. As you know, MDA’s commitment to improving the lives of young adults living with muscle-debilitating diseases is near to my heart. It’s why I chose to accept the role as the first adult MDA National Goodwill Ambassador. It’s been almost a year since we began implementing MDA’s . . .
For most, a trip to Target is just a way to cross off another item on a lengthy to-do list, or a quasi-addictive way to reduce stress via retail therapy. For 14-year-old Ethan Pyles and his mother, Sandra, a trip to Target brought renewed hope, grateful hearts, and optimism for thousands of boys who have . . .
MDA and RYR-1 Foundation have announced a partnership aimed at advancing research and clinical care, raising awareness and improving education of patients, medical professionals and the public about RYR1-related myopathies. The partnership represents a key step in MDA’s commitment to form collaborative relationships with other organizations working on the same diseases MDA covers. “MDA is . . .
MDA resource specialist Mandy Behling used to think that living unlimited meant swimming with the sharks and climbing Mt. Everest. However, she has come to find that living unlimited means more than just traveling the world and seeking out grandiose adventures.
“Now I think that living unlimited means pushing myself just a little harder than I thought possible the day before,” she says.
If there was one piece of advice MDA Resource Specialist Ashleigh Peska could offer individuals with muscular dystrophy and other muscle-debilitating diseases, it would simply be, “don’t quit!” Ashleigh, who lives with limb-girdle muscular dystrophy (LGMD), is a shining example of this mantra. A college graduate and former MDA Iowa State Goodwill Ambassador, Ashleigh has . . .
On Sept. 19, the U.S. Food and Drug Administration granted accelerated approval to eteplirsen, which will now be marketed under the brand name Exondys 51 for the treatment of some forms of Duchenne muscular dystrophy (DMD). We’ve received a lot of questions about the newly approved drug, and wanted to share a few answers to . . .
In a joint statement today, Biogen and Ionis Pharmaceuticals announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Submission of the NDA is the first step toward getting approval . . .