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An expression of the live unlimited spirit, stories and voices of the MDA community

Santhera Receives FDA Fast Track Designation for Treatment of Duchenne Muscular Dystrophy

Press Release: Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystrophy Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “This is exciting news for our community. MDA is committed to bringing safe and effective treatments and cures to kids and adults living with neuromuscular . . .

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BioBlast Pharma Receives FDA Fast Track Designation for Treatment of Oculopharyngeal Muscular Dystrophy

Press Release: BioBlast Pharma Announces Granting of Fast Track Designation by the FDA for Cabaletta in Oculopharyngeal Muscular Dystrophy (OPMD) Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “We are thrilled to see this continued progress from the FDA. OPMD is a rare and debilitating muscular dystrophy for which there . . .

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MDA to Offer Genetic Testing for Limb-Girdle Muscular Dystrophy

MDA, Genzyme and Emory University team up to expand access to genetic testing for accurate diagnosis of limb-girdle muscular dystrophy CHICAGO, March 26, 2015 — The Muscular Dystrophy Association announced today that thanks to a grant from Genzyme, a Sanofi company, and in collaboration with Emory Genetics Laboratory, MDA clinics are now offering genetic testing . . .

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MDA Scientific Conference Update: March 14 Morning Sessions

Saturday morning’s presentations were devoted to the latest news about clinical trials and drug development. Among the highlights: Duchenne muscular dystrophy (DMD): Edward Kaye from Sarepta Therapeutics presented his company’s most recent data about its experimental drug eteplirsen, designed to treat about 13 percent of the Duchenne muscular dystrophy (DMD) population, and discussed plans for . . .

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MDA Scientific Conference Update: March 13 Afternoon Sessions

Friday’s afternoon sessions focused on laboratory studies in animal and cell models of neuromuscular diseases. These kinds of studies are necessary before treatments can be tested in humans. Among the highlights: Spinal muscular atrophy (SMA): Brian Kaspar from Nationwide Children’s Hospital in Columbus, Ohio, presented experiments from his lab to develop gene transfer therapy for . . .

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MDA Scientific Conference Update: March 13 Morning Sessions

Friday’s morning sessions focused on deepening understanding of the mechanisms underlying diseases of the muscles and nervous system Among the highlights: Spinal muscular atrophy (SMA): Christine DiDonato from Northwestern University presented her work with mice that have a disease mimicking spinal muscular atrophy (SMA) with varying degrees of severity, which is characteristic of human SMA . . .

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MDA Underscores Commitment to Frontline Discovery, Breakthroughs at Research Conference

CEO asks physician-scientist attendees to embrace the urgency to find treatments and cures and join MDA as part of bold plan WASHINGTON, March 12, 2015 – The Muscular Dystrophy Association’s chief executive today opened its 2015 Scientific Conference in Washington by declaring to the more than 400 physician-scientist attendees that MDA’s number one mission priority . . .

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MDA Scientific Conference Update: March 12 Afternoon Sessions

Thursday’s afternoon sessions focused on therapeutic approaches now in development Here are some highlights: Duchenne muscular dystrophy (DMD): Dean Burkin from the University of Nevada, Reno, presented his team’s findings that show how raising levels of a protein called alpha 7 beta 1 integrin has potential as a therapy for treating Duchenne muscular dystrophy. A . . .

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MDA Scientific Conference Update: March 12 A.M. Sessions

Thursday morning focused on understanding the genetics and the “epigenetics” of neuromuscular disorders Epigenetics is the relatively new field of factors that influence the activity of genes but do not change the basic coding (DNA sequence) of a gene. Identifying the genetic and epigenetic pathways of a disease can allow for better diagnosis and genetic . . .

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