MDA and RYR-1 Foundation Team Up in the Fight Against RYR1-Related Myopathies

MDA and RYR-1 Foundation have announced a partnership aimed at advancing research and clinical care, raising awareness and improving education of patients, medical professionals and the public about RYR1-related myopathies. The partnership represents a key step in MDA’s commitment to form collaborative relationships with other organizations working on the same diseases MDA covers. “MDA is . . .

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Meet the MDA Resource Specialists: Mandy

MDA Resource CenterMDA resource specialist Mandy Behling used to think that living unlimited meant swimming with the sharks and climbing Mt. Everest. However, she has come to find that living unlimited means more than just traveling the world and seeking out grandiose adventures.

“Now I think that living unlimited means pushing myself just a little harder than I thought possible the day before,” she says.

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Meet Ashleigh: MDA Resource Specialist

If there was one piece of advice MDA Resource Specialist Ashleigh Peska could offer individuals with muscular dystrophy and other muscle-debilitating diseases, it would simply be, “don’t quit!” Ashleigh, who lives with limb-girdle muscular dystrophy (LGMD), is a shining example of this mantra. A college graduate and former MDA Iowa State Goodwill Ambassador, Ashleigh has . . .

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Top 5 FAQs: Eteplirsen (Exondys 51) for DMD Treatment

On Sept. 19, the U.S. Food and Drug Administration granted accelerated approval to eteplirsen, which will now be marketed under the brand name Exondys 51 for the treatment of some forms of Duchenne muscular dystrophy (DMD). We’ve received a lot of questions about the newly approved drug, and wanted to share a few answers to . . .

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Biogen Submits New Drug Application for Nusinersen to Become an Approved Treatment for SMA

In a joint statement today, Biogen and Ionis Pharmaceuticals announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Submission of the NDA is the first step toward getting approval . . .

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MDA Moves Newborn Screening Forward in Partnership with Cure SMA

This September as we recognize Newborn Screening Awareness month, we are proud to announce a new partnership with Cure SMA aimed at moving newborn screening forward for spinal muscular atrophy (SMA)—the leading genetic cause of death in infants. This is an exciting time for the neuromuscular disease community as decades of research are translating into . . .

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Catalyst Pharmaceuticals’ MG Drug Gets FDA Orphan Drug Designation

The investigational drug amifampridine phosphate (brand name Firdapse), under development by Catalyst Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Orphan Drug designation for the treatment of myasthenia gravis (MG). Amifampridine phosphate is a potassium channel inhibitor designed to prolong signals released from nerves and allow greater stimulation of muscles. The drug currently is . . .

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NIH Seeks Children and Caregivers to Participate in Quality of Life Study

Researchers at the National Institutes of Health (NIH) seek children with neuromuscular disorders (NMDs) and/or their caregivers (parent or legal guardian) to complete questionnaires. Researchers would like to develop a tool to better assess the quality of life of people with NMDs, such as muscular dystrophy. The goal of this study is to validate two different . . .

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Eteplirsen Granted Approval for DMD: Turning Promise into Progress

Today we couldn’t be happier as, together with the Duchenne community, we celebrate FDA accelerated approval of eteplirsen to treat kids and adults with some forms of Duchenne muscular dystrophy (DMD). “This is something that we’ve watched, and hoped and prayed for, and seen it develop over the last seven years or so,” said Josh . . .

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