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Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July…

How these promising therapies are being studied and tested for neuromuscular diseases

At the 2019 MDA Clinical & Scientific Conference, one scientific session of interest centered on what’s new in neuromuscular diseases (NMDs). In this session, presenting scientists discussed progress in therapy…

Healthcare providers who work with patients diagnosed with neuromuscular disease (NMD) must consider the impact of these diseases on cognition and behavior, said presenters at a 2019 Clinical & Scientific…

During a late-morning session on the final day of the 2019 Clinical & Scientific Conference, five healthcare professionals — Laurie Sterling, a speech therapist at the Texas Children’s Hospital; Dr.…

A young man with SMA answers this question by becoming a student leader

Everyone is born, and everyone dies. What happens in between — colds and flu, cardiac problems, cancer, amyotrophic lateral sclerosis (ALS) — is anyone’s guess. And people do guess. It’s common — indeed, almost…

After two years of laying the groundwork, the Agency for Toxic Substances and Disease Registry (ATSDR) has opened its National ALS Registry to collect information from people living with ALS. Scientists…

Recently, media outlets reported a possible link between developing ALS and living near Lake Mascoma in Western New Hampshire. The Union Leader in New Hampshire and other news outlets indicated that the…

A government-supported research team announced in the Sept. 23 issue of the journal Neurology that having served in southwest Asia during the Gulf War significantly increases the risk of later…