The U.S. Food and Drug Administration (FDA) in August accepted Marathon Pharmaceuticals’ New Drug Application for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. A decision on deflazacort, which previously has received Fast Track status, Orphan Drug designation and Rare Pediatric Disease status from the FDA, is anticipated in February . . .
Muscular dystrophy is a term that refers to a number of diseases that cause progressive loss of muscle mass resulting in weakness and, sometimes, loss of mobility. There are many different kinds of muscular dystrophy, each affecting different groups of muscles. In some types of muscular dystrophy, symptoms begin in childhood. In other forms, symptom . . .
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 . . .
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 . . .
Researchers are seeking participants for a trial designed to assess the safety and feasibility of a home-based exercise program in boys with Duchenne muscular dystrophy (DMD). Prior studies in milder forms of muscular dystrophy and in rodent models suggest that resistance exercise may have beneficial effects for maintenance of muscle mass in DMD, but data . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
