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This year has been an eventful year for newborn screening in neuromuscular disease, with potential further progress on the near horizon. We began 2025 with all 50 states screening for…

For many families with kids, the end of summer includes preparations for heading back to school for a new year of growth, learning, and activities. On top of school supplies,…

Researchers at Solid Biosciences are seeking boys with Duchenne muscular dystrophy (DMD) to participate in a phase 1/2 clinical trial (INSPIRE DUCHENNE) to evaluate the safety and efficacy of the investigational…

Find answers to the five most common corticosteroid questions as well as other considerations if you’re prescribed them to treat a neuromuscular disease.

My name is Santana Gums and I have LGMD-2b. I live in Arizona and work as a Legal Assistant. Back in February of this year, I attended the Arizona Muscle…

Researchers at Albany Medical College are seeking parents and caregivers of children with Duchenne muscular dystrophy (DMD) to participate in an observational REDCap survey about their adherence to DMD care guidelines…

Charcot-Marie-Tooth disease (CMT) encompasses a group of inherited disorders that affect movement and sensation in the arms, legs, hands, and feet. Common symptoms include lower leg weakness, foot deformities, reduced…

Researchers at the Center for Health + Technology at the University of Rochester are seeking individuals with non-dystrophic myotonias (NDM) to participate in an observational interview. The findings of the interviews…

While every day is a good day to enjoy the simple pleasure of reading, National Read a Book Day on September 6th offers a fun reminder for booklovers to slow…

Dr. Kaitlin Batley, Engage Steering Committee member, MDA Care Center Director, Assistant Professor at UT Southwestern, and neurologist at Children’s Health. As MDA and the Engage Steering Committee gear up…