From Summer Camp to Chicago Marathon, Robb Valentyn Goes All in for MDA
From MDA Summer Camp to MDA Team Momentum, Robb Valentyn has been volunteering with MDA for ten plus years and doesn’t plan to stop anytime soon.
Strongly
An expression of the live unlimited spirit, stories and voices of the MDA community
Promising results in phase 1 SMA gene therapy trial
Chicago-based biotechnology company AveXis has announced encouraging results from its completed phase 1 gene therapy trial to test the investigational therapy AVXS-101 in infants with type 1 SMA.
What is Advocacy and Why Should You Care?
In advance of MDA’s first ever Advocacy Conference, taking place April 23-25, we asked several advocates to unpack the idea of advocacy: what is it, why they became advocates and why it is necessary for others to follow their examples. Their responses follow.
High-School Track Athlete with Myotonic Dystrophy Finds His Stride Helping Others
Almost exactly one year ago, in the spring of 2016, Kade McCann learned he had myotonic dystrophy. For this young athlete, the diagnosis stopped him in his tracks.
Encouraging Trial Data Reported for ALS Drug Masitinib
AB Science has reported encouraging top line trial results from its completed phase 2/3 study in Europe of the experimental drug masitinib in ALS. Additional testing is likely required, but if successful, masitinib could one day become an approved therapy for ALS.
U.S. Air Force Senior Airman runs with Team Momentum for his family and all MDA families
Air Force Senior Airman Mike Hall, 20th Aerospace Medicine Squadron flight and operation medical technician conquered the 2017 Boston Marathon with Team Momentum, running for his sister Danielle, in memory of his brother DJ and for all families impacted by neuromuscular diseases.
I’m an Adult. Talk to Me Like One.
I’m a 25-year-old college graduate. I’m an adult and I can prove it. I have bills, grown-up responsibilities and the obligation to do things I don’t want to do. Still, regardless of the date of birth listed on my Colorado State ID, some people still treat me like a kid. Unfortunately, a lot of the world finds it easy to infantilize people with disabilities.
MDA Cohosts and Sends Representatives to SMA Patient Focused Drug Development Meeting with FDA
Being chosen to speak in front of the FDA and represent the SMA community makes me feel incredibly honored. Today’s SMA Patient-Focused Drug Development (PFDD) meeting with the FDA means we have a voice as patients living with rare muscle diseases. The PFDD meeting was set up so that patients with SMA and the parents of children with SMA could stand before the FDA and voice their concerns, discuss their experiences with research and address the need for continued research that evolves to include more patients. This is my story.
The Powerhouse With the Power Chair: Meet Kierra, MDA’s North Carolina State Ambassador
She’s always been a whirlwind. But Kierra’s new power chair has turned that whirlwind into a tiny tornado.
Trial Seeks Participants to Test CK-2127107 in SMA
Investigators are seeking participants for an ongoing phase 2 clinical trial, sponsored by Cytokinetics, to test the investigational drug CK-2127107 in people with types 2, 3 or 4 SMA.
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