Alexion Seeks Approval of Soliris as a Treatment for MG
Alexion Pharmaceuticals announced it has submitted a marketing application to the FDA for the investigational drug eculizumab (brand name Soliris).
Category: News
FDA Approves First-Ever Treatment of SMA
The U.S. Food and Drug Administration (FDA) has approved Biogen’s nusinersen (brand name Spinraza) for the treatment of spinal muscular atrophy (SMA).
A Year of Progress and Promise
MDA began 2016 with an ambitious blueprint, full of changes and improvements — all designed to help us fulfill our mission promise to free individuals and families from the harmful effects of muscular dystrophy and related life-threatening diseases. Now, we reflect on our accomplishments and give thanks to our supporters.
CMS Study Seeks Participants
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.
Encouraging Clinical Study Data Reported for ALS Drug Edaravone
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 . . .
MDA Holiday Gift Guide
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 . . .
Introducing MDA’s Revamped Programs for Young Adults with Neuromuscular Diseases
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 . . .
A Clinical Trial, a Trip to Target and the Approval of Exondys 51
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 Team Up in the Fight Against RYR1-Related Myopathies
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 . . .
Meet the MDA Resource Specialists: Mandy
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.
