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.

Read More

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.

Read More

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 . . .

Read More

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 . . .

Read More

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 . . .

Read More

Meet the MDA Resource Specialists: Mandy

MDA Resource CenterMDA 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.

Read More