When I was asked to continue in my role as National Ambassador for MDA in 2017, I had to think about it for about a nanosecond before I gave my answer. Yes, of course! So it’s official: I’ve re-upped for another tour. 2016 will be a hard act to follow, but now that I’m a registered frequent flyer and have a year of ambassadorship under my belt, I can’t wait to try to top it. In fact, I’ve taken the liberty of compiling a list of experiences I’m looking forward to in 2017. Join me, won’t you?
On Dec. 23, 2016 the FDA approved Spinraza for the treatment of SMA. In clinical trials to test Spinraza, participants who received treatment with the drug experienced life-changing outcomes they wouldn’t have been expected to achieve.
Strength, Science, and Stories of Inspiration (SSSI) and the Muscular Dystrophy Association (MDA) announced a new research funding mechanism for graduate students and postdoctoral trainees working in the muscle disease field.
MDA is excited to announce that we are accepting applications for the inaugural National Community Advisory Committee, a group that will advise MDA on matters of importance to families living with neuromuscular diseases and help inform MDA’s efforts to support individuals to live unlimited.
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.