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features: Neuromuscular Disorders

FDA grants accelerated approval for SRP-9001 as the first ever gene therapy for Duchenne muscular dystrophy

On 22nd June, 2023, the U.S Food and Drug Administration (FDA) granted accelerated approval to Sarepta Therapeutics’ SRP-9001 (ELEVIDYS, delandistrogene…

First ever targeted treatment for SOD1-ALS: FDA approve tofersen

On April 25th, 2023, the U.S. Food and Drug Administration (FDA) approved tofersen (BIIB067) for the treatment of amyotrophic lateral…

Newborn screening improves health outcomes in infants with spinal muscular atrophy

Spinal muscular atrophy (SMA) is a severe genetic disease associated with mutations in the SMN1 gene (SMA5q). The pathological characteristics…

Top clinical trial updates from AAN 2021: MG, PD & ALS

Discover updates from major clinical trials in myasthenia gravis, Parkinson’s disease & ALS presented at AAN 2021!