Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) is continuing review and internal discussions related to its New Drug Application for eteplirsen and will not complete the work by the goal date of May 26. Eteplirsen is an exon skipping drug designed to slow the progression of Duchenne muscular dystrophy (DMD) . . .
Dear MDA family and friends, As part of MDA’s commitment to provide our DMD community with up-to-date research and advocacy news, we want to let you know that MDA today made another appeal to the U.S. Food and Drug Administration in support of eteplirsen, which, if approved, could help slow progression of the disease for . . .
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 . . .
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 . . .
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. . . .
Researchers are looking for boys with Duchenne muscular dystrophy (DMD) to participate in the ongoing phase 3 clinical trial, Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD). The trial will compare three different corticosteroid regimens in boys with DMD, ages 3 to 7 years. Corticosteroids, such as prednisone and deflazacort, work as anti-inflammatories or . . .
Ryan Hometown: Naperville, IL Age: 13 Diagnosis: I have Duchenne Muscular Dystrophy, which is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Favorite School Subject/Activities: My favorite school subjects are PE and Math. Favorite People and/or Pets: Aside from my mom, dad, and sisters, I love the Chicago Blackhawks. Interests: I enjoy going to watch . . .
MDA Muscle Walk is a life-changing event that strengthens families and communities. But it’s more than anotherfundraising walk. It’s a powerful experience that forges lifelong connections, celebrates families and the barriers they overcome and turns hope into answers. We will be featuring stories of MDA families and their dedication to Muscle Walk here on Strongly . . .
MDA recognizes the importance of early diagnosis for neuromuscular disorders to optimize opportunities for effective treatment. This includes supporting initiatives to understand the impact of newborn screening in neuromuscular disorders. In early March, MDA sponsored an international conference in St. Louis, MO, “Newborn Screening for Neuromuscular Diseases: Improving Patient Outcomes and Evaluating Public Health Impact,” that . . .
