Neuromuscular Disease News

Patients with neuromuscular diseases face an increased risk for respiratory muscle weakness, which can contribute to various health and mental problems that take a significant toll on their lives.

Regenxbio Inc. announced that the FDA has granted fast track designation for RGX-202, a one-time gene therapy for treatment of Duchenne muscular dystrophy.

Vico Therapeutics announced that the first patient has been dosed in a phase 1/2a study evaluating VO659, an investigational antisense oligonucleotide for Huntington’s disease and spinocerebellar ataxia types 1 and 3.

Hyperbaric oxygen therapy improved pain symptoms, quality of life and social and emotional function in adults with fibromyalgia after traumatic brain injury compared with pharmacologic intervention, according to research in PLOS One.

Prilenia Therapeutics announced the completion of its global phase 3 study of pridopidine for Huntington’s disease, with topline results expected in the next few months.

Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Viltolarsen delayed disease progression over 4 years in children with Duchenne muscular dystrophy, with most treatment-emergent adverse events being mild or moderate, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Ataluren preserved muscle burst activity across a range of ambulatory tests compared with placebo in boys with nonsense mutation Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Ataluren preserved upper limb function in boys with nonsense mutation Duchenne muscular dystrophy, according to a poster presented at the MDA Clinical & Scientific Conference.

Treatment with casimersen increased exon 45 skipping from baseline and was well-tolerated in children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.