Strongly


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

Encouraging Results in Phase 1 CALLISTO Trial to Test Omigapil in CMD

A phase 1 trial to test omigapil in congenital muscular dystrophy (CMD) conducted at the National Institutes of Health is complete and data indicate it met its primary objective. Results from the trial, also known as CALLISTO, show a favorable pharmacokinetic profile of omigapil and demonstrated that the drug was safe and well-tolerated in children and . . .

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Catalyst Pharmaceuticals Submits New Drug Application to FDA for Firdapse to Treat LEMS

Catalyst Pharmaceuticals has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug amifampridine phosphate (brand name Firdapse) for the treatment of people with Lambert-Eaton myasthenic syndrome (LEMS). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. If the FDA . . .

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2018 MDA Clinical Conference Highlight Report: Day 2 Afternoon Sessions

The Tuesday afternoon sessions of MDA’s 2018 Clinical Conference focused on best practices in newborn screening. Augmentative communication, pediatric-to-adult transition, respiratory health and care coordination also were covered in parallel sessions. Among the highlights: Newborn screening R. Rodney Howell, M.D., from the Miller School of Medicine, University of Miami, talked about the history of newborn . . .

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2018 MDA Clinical Conference Highlight Report: Day 2 Morning Sessions

On Tuesday morning, MDA’s 2018 Clinical Conference sessions focused on best practices in cardiac care, physical therapy, bone health, technology and nutrition. Among the highlights: Cardiac management for the muscular dystrophy lifespan Elizabeth McNally, M.D., Ph.D., from Northwestern University Feinberg School of Medicine, noted that to best manage the heart in neuromuscular disease, a genetic . . .

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2018 MDA Clinical Conference Highlight Report: Day 1 Afternoon Sessions

Monday’s afternoon sessions at MDA’s 2018 Clinical Conference focused on advances in personalized medicine, including the latest in precision therapies, genomics, digital health and MDA’s MOVR initiative. Among the highlights: Correction of Duchenne muscular dystrophy by genome editing Leonela Amoasii, Ph.D., from UT Southwestern Medical Center, discussed a vision for gene editing in DMD. The . . .

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2018 MDA Clinical Conference Highlight Report: Day 1 Morning Sessions

Monday’s morning sessions at MDA’s 2018 Clinical Conference focused on what’s new in ALS (amyotrophic lateral sclerosis), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy (DM) and myasthenia gravis (MG). Among the highlights: ALS  James D. Berry, M.D., M.P.H., from Massachusetts General Hospital, noted that, “The really profound answer to what’s new in . . .

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Hope is Here: MDA Blogger Chris Anselmo Recaps Our 2018 Clinical Conference

Last week, I had the opportunity to attend the 2018 MDA Clinical Conference in Arlington, Va., to write about it from the patient’s perspective. I am always excited to attend conferences that bring together stakeholders in the neuromuscular disease community. I love meeting fellow patients, and I especially love learning about the latest scientific and . . .

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