Genetic Testing Provides Answers and Hope

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 . . .

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Five Questions with ALS Researcher Asim Beg

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 . . .

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A Letter to Our Community About the Eteplirsen Advisory Committee Outcome and Next Steps

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 . . .

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Testimony from MDA at FDA Advisory Committee Hearing for DMD Drug Eteplirsen

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 . . .

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Eteplirsen Advisory Committee Meeting: Background Materials and Webcast Information

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 . . .

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With a Little Help from Kevan’s Friends

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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Meet Devin, MDA’s 2016 State Goodwill Ambassador from Wisconsin

Devin Hometown: Manitowoc, Wisconsin Diagnosis: Duchenne muscular dystrophy Age: 14 Favorite School Subject/Activities: I really like science and health. Favorite People and/or Pets: I love my family, my dogs and everyone at MDA. Interests: I enjoy drawing cars, watching sports, riding ATVs, making hand-made rainbow loom bracelets and playing video games. My favorite Things About MDA: MDA helps so many people. . . .

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