Strongly


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

Clinical Trial Alert: Phase 3 Study of Pamrevlumab (FG-3019) in Combination with Systemic Corticosteroids in Ambulatory Boys with DMD

Researchers at FibroGen are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial to evaluate efficacy of the investigational drug pamrevlumab (FG-3019) in combination with systemic corticosteroids to treat DMD. Pamrevlumab is designed to decrease fibrosis in the muscles, thereby protecting the heart and lungs. This has the potential . . .

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Clinical Trial Alert: Phase 2 Study of Pitolisant for Treatment of Excessive Daytime Sleepiness in Adults with Myotonic Dystrophy, Type 1 (DM1)

Researchers at Harmony Biosciences are seeking adults living with myotonic dystrophy, type 1 (DM1) to participate in a phase 2 clinical trial and open-label extension to evaluate safety and efficacy of the investigational drug pitolisant (brand name Wakix) to treat excessive daytime sleepiness and other non-muscular symptoms of DM1. Pitolisant treatment increases the amount the chemical . . .

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Clinical Trial Alert: Phase 1b and Open-Label Extension Study of ASP0367 in Boys with DMD

Researchers at Astellas Pharma Inc. are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial and open-label extension to evaluate safety, tolerability and preliminary efficacy of the investigational drug ASP0367 (MA-0211) to treat DMD. ASP0367 is designed to increase the number and function of the mitochondria in cells, thereby . . .

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Spotting Neuromuscular Disease in Adults

After years of denial, it was the stairs to his second-floor college apartment that forced Chris Anselmo to finally face his limb-girdle muscular dystrophy (LGMD) in 2009. “I remember moving in, trying to carry things like my desk chair to the top of the stairs, and just feeling completely weak and exhausted by the time I reached . . .

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Inclusion in STEM: A Discussion with General Motors

According to the National Science Foundation, individuals living with a disability, such as neuromuscular disease, are underrepresented in the fields of science, technology, engineering, and math (STEM). Yet STEM-based careers are growing exponentially, and diversity is needed to drive innovation in these fields. As part of MDA’s mission to transform the lives of individuals living with neuromuscular . . .

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Biogen’s Early Access Program for Tofersen is Now Open for Individuals with Rapidly Progressing SOD1-ALS

Beginning in mid-July 2021, Biogen is opening part 1 of its early access program for the investigational therapy tofersen to individuals with amyotrophic lateral sclerosis (ALS) caused by mutation of the superoxide dismutase 1 gene (SOD1) who are specifically experiencing rapid disease progression. Tofersen is being investigated in a phase 3 clinical study for safety and efficacy, and the . . .

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Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Adults with Generalized Myasthenia Gravis

Researchers at The Neurological Institute, PA, located in Charlotte, North Carolina, are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 3 clinical trial to evaluate efficacy and safety of the investigational drug rozanolixizumab. Rozanolixizumab is designed to alter the immune response and reduce antibody production, potentially improving strength, fatigue, swallowing, and . . .

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National Connections Program Helps MDA Families Find Others with Shared Experiences

When MDA launched the MDA National Connections program last year, there was no doubt it would be invaluable for fostering relationships, especially during the pandemic.  It didn’t take long to see that the MDA community was eager to build connections, with interest coming from families and people of all ages, from parents of children to individuals . . .

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