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.
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.
For more than 60 years, firefighters have teamed with MDA to fight back against muscular dystrophy, ALS and related life-threatening diseases.
For one firefighter, the cause became very personal when his son, Chandler Mallen, was diagnosed with juvenile dermatomyositis, a neuromuscular disease that involves inflammation of the muscles. When Chandler asked his father — Scott, a firefighter who has collected donations for MDA’s Fill the Boot program for almost 25 years — if he’d lose the use of his legs, he didn’t wait for an answer.
“Even if I do,” Chandler said, “I’ll never stop trying.”
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 . . .
When I was 25, I took an unlikely job as an editor at a travel magazine. As a journalism graduate, the editing part of the job was a natural fit; as someone who’d never flown on a plane, the traveling part seemed a little daunting. I’d been diagnosed with Charcot-Marie-Tooth disease six years prior, but . . .
Gianna Schwartz was born to write. Just 10 years old, the fifth-grader from Bettendorf, Iowa, has already won writing contests sponsored by PBS and participated in poetry slams. She’s even written a children’s book that was purchased at an auction by a Major League baseball player. Gianna — who is the Iowa State Goodwill Ambassador . . .
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 . . .
Results from the STEADFAST phase 3 clinical trial in Friedreich’s ataxia (FA) failed to show that the drug ACTIMMUNE® (interferon gamma-1b) was effective against any of the study’s disease measurements, according to Horizon Pharma. The study hoped to slow disease progression as measured by a functional rating scale assessing capacities such as speech, ability to . . .