Strongly


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

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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Clinical Trial Alert: Phase 3 Study of ION363 in Individuals with ALS with Fused in Sarcoma Mutations (FUS-ALS)

Researchers at IONIS Pharmaceuticals are seeking individuals living with amyotrophic lateral sclerosis (ALS) with fused in sarcoma mutations (FUS-ALS) to participate in a phase 3 clinical trial to determine the efficacy of the investigational drug ION363. Drug efficacy will be determined by measuring clinical functioning and survival in trial participants.   The study consists of two parts, . . .

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The Search for ALS Treatments Continues

May is Amyotrophic Lateral Sclerosis (ALS) Awareness Month, and while this past year was different — given the global pandemic we all endured — the research and support for finding new treatments never stopped.  ALS is a neurodegenerative disease of the motor neurons that eventually causes muscular atrophy, paralysis, and death. The cause of ALS is understood . . .

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Help and Hope for Friedreich’s Ataxia

Bella, a 15-year-old in Fort Worth, Texas, loves jellyfish, horses, anime, Billie Eilish, reading sci-fi and fantasy, and making art. But living with Friedreich’s ataxia (FA) makes it hard for her to engage in some of her favorite activities. “I can draw and paint for only a few minutes before my hand starts to hurt, so I . . .

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Research Study Alert: Survey for Individuals with DMD — The Duchenne Muscular Dystrophy Health Index (DMD-HI)

Researchers at University of Rochester are seeking individuals living with Duchenne muscular dystrophy (DMD) to participate in a survey to help with development and validation of a disease-specific, patient-reported outcome measure for clinical trials involving people with DMD. Participants will need to complete an online survey and a follow-up phone interview. The survey will take approximately 20 . . .

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