Strongly


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

Clinical Trial Alert: Amicus Therapeutics Seeks Teenage Participants for a Phase 3 Late-Onset Pompe Disease Study

Researchers at Amicus Therapeutics are looking for adolescents (age 12-18) with late-onset Pompe disease (LOPD) to participate in a phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care. Participants will be . . .

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Casey’s General Stores Round Up for MDA

This September, at Casey’s General Stores in 16 states across the Midwest, employees have been doing more than serving guests. They’ve been serving the MDA community, too, inviting store guests to round purchase totals up to the nearest whole dollar to support individuals and families impacted by neuromuscular disease. Now in its 13th year of . . .

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New MDA Educational Program Offers a 360-Degree View of ALS Care

MDA’s new Medical Education department was established in response to the growing need for clinician education in neuromuscular diseases. We are pleased to present our first program highlighting amyotrophic lateral sclerosis (ALS): a PowerPoint slide deck. ALS is a motor neuron disease that leads to the widespread loss of muscle use. Patients diagnosed with ALS . . .

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2019 MDA Engage DMD Symposium Scheduled on Nov. 9

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Duchenne muscular dystrophy (DMD), as well as their caregivers and loved ones, to attend the MDA Engage DMD Symposium taking place Nov. . . .

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2019 MDA Engage Charcot-Marie-Tooth Symposium Scheduled on Nov. 9

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Charcot-Marie-Tooth disease (CMT), as well as caregivers and loved ones, to attend the MDA Engage CMT Symposium taking place Nov. 9 in . . .

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Join Us for a Virtual MDA Engage BMD Symposium on Oct. 26

We invite you to attend an upcoming virtual MDA Engage Becker Muscular Dystrophy Symposium where we will share information from experts in the field about Becker muscular dystrophy (BMD).  The online event will take place Oct. 26 from noon-4 p.m. ET. Event chair Dr. Paula Clemens from the University of Pittsburgh School of Medicine and . . .

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NS Pharma Submits New Drug Application to FDA for Viltolarsen to Treat DMD Amenable to Exon 53 Skipping

On Oct. 2, NS Pharma Inc. (a wholly-owned US subsidiary of Nippon Shinyaku Co. Ltd.) submitted a rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its exon-skipping therapy under development, viltolarsen (previously NS-065/NCNP-01), to treat Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. On Sept. 26, Nippon Shinyaku . . .

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Amy and Jamie Shinneman Find Purpose on Their Way to the Bank of America Chicago Marathon

For the last four months, Amy Shinneman has been training to do something she never imagined doing: hitting the pavement as a participant in the Bank of America Chicago Marathon. Amy, a 45-year-old Noblesville, Ind., native, lives with Bethlem myopathy, a form of Ullrich congenital muscular dystrophy that causes muscle weakness throughout her limbs. “From . . .

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