An expression of the live unlimited spirit, stories and voices of the MDA community
Christine Vande Velde, associate professor at the University of Montreal CHUM Research Center in Montreal, Quebec (Canada), was awarded an MDA research grant totaling $300,000 over a period of three years to study what goes wrong in amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene. In ALS patients with . . .
When Kathy Payette was in eighth grade, she started experiencing unexplained muscle weakness. Over a period of three years in the mid-1970s, she visited several doctors, all of whom were puzzled by her symptoms. When she was 17, Kathy spent a week at the Mayo Clinic with a team of doctors who concluded she had . . .
Asim Beg, assistant professor at University of Michigan in Ann Arbor, was awarded an MDA research grant totaling $300,000 over a period of three years to study the role of a protein, EphA4, in amyotrophic lateral sclerosis (ALS). High levels of EphA4 correlate with rapid disease progression in ALS patients. Beg and colleagues will work . . .
Dear MDA family and friends, At yesterday’s all-day FDA advisory committee meeting for the promising drug eteplirsen, MDA stood shoulder to shoulder with our sister groups, research and clinical experts, and most importantly you, our families. We couldn’t have been more proud to raise our voice on behalf of the thousands of Duchenne muscular dystrophy . . .
Today, MDA’s Executive Vice President & Chief Medical and Scientific Officer Dr. Valerie Cwik spoke during the FDA’s Advisory Committee hearing to review eteplirsen, under development by Sarepta Therapeutics for the treatment of some forms of Duchenne muscular dystrophy (DMD). Good afternoon. I am Dr. Valerie Cwik, and I am pleased to be here today on behalf . . .
For Cheryle Elliot and her son, Dakota, the Muscular Dystrophy Association is like a part of the family. From selling MDA Shamrocks to attending their local MDA Lock-Up event to participating in Muscle Walk, they do it all — with Dakota smiling and laughing all the way through. Dakota was born with congenital myotonic muscular . . .
The FDA has made available background materials and webcast information for the advisory committee meeting to review eteplirsen on Monday, April 25. To view the complete set of background information including a meeting agenda, meeting roster, committee roster and briefing information, visit 2016 Meeting Materials, Peripheral and Central Nervous System Drugs Advisory Committee. The Center . . .
Marley Hometown: Mystic, CT Diagnosis: I have Spinal Muscular Atrophy Type 2, and I was diagnosed when I was 1 year old. SMA is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Age: 15 Favorite School Subject/Activities: I am currently a sophomore in high school. My favorite subjects . . .
Andrew Hussey runs to work and he runs home from work. He pounds the pavement on the weekends. He runs upwards of 80 miles a week — more than 600 miles since January — training for the multiple races he’ll run this year. But he doesn’t run all those miles for himself. He runs for . . .
When Kevan Chandler and his friends decided to go backpacking in Europe this summer, they expected a few dozen friends and family members to be interested in their plans. Little did they know their adventure would become international news. The media response to the trip has been “awesome” according to Kevan. He and his travelling . . .
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