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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First Appeal for FDA to Review Translarna Denied

PTC Therapeutics shared the latest news today on efforts to make its experimental drug Translarna available in the U.S. for the treatment of some forms of Duchenne muscular dystrophy (DMD). The company reported that the U.S. Food and Drug Administration (FDA) has denied its first appeal that a New Drug Application for Translarna be accepted . . .

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Recap: Our 2016 Becker Muscular Dystrophy Conference

On Sept. 24, we held our annual Becker Muscular Dystrophy (BMD) Conference aimed at connecting families with researchers and other individuals living with BMD. The conference took place in Washington, D.C., and featured a wide range of topics that included understanding the root causes of BMD, how genetics and other factors contribute to the variation . . .

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Five Questions with Researcher Madhuri Hegde

Madhuri Hegde, associate professor in the department of human genetics at Emory University in Atlanta, was awarded an MDA research infrastructure grant totaling $300,000 over a period of three years to continue groundbreaking work to identify and characterize new gene defects that can cause limb-girdle muscular dystrophy (LGMD). In the era of precision medicine, it . . .

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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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MDA and Target ALS Will Work Together to Support Young ALS Investigators

MDA and Target ALS Foundation have announced a partnership aimed at advancing ALS research and therapy development. Through the partnership, the two organizations will work together to support talented young scientists who choose to pursue a career devoted to ALS research. The partnership represents a key step in MDA’s commitment to form strategic alliances with . . .

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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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Five Questions with Friedreich’s Ataxia Researcher Michael Huang

Michael Huang, the NHMRC Peter Doherty Postdoctoral Fellow in the department of pathology, Bosch Institute, at the University of Sydney, Australia, was awarded an MDA development grant totaling $177,100 over a period of three years to explore how deficiency of the frataxin protein in Friedreich’s ataxia (FA) may alter the function of cellular power supplies . . .

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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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