Spinal Muscular Atrophy

Treatment with nusinersen was linked to increased motor function and reduction of the neurodegenerative biomarker neurofilament light chain in children with spinal muscular atrophy, according to interim results of a study.

Treatment with apitegromab was linked to improved muscle strength and range of motion, while being safe and well-tolerated in those with spinal muscular atrophy types 2 and 3, according to a poster presentation.

Treatment with Zolgensma improved motor function in a cohort of children with spinal muscular atrophy, according to a poster at the 2024 MDA Clinical & Scientific Conference.

Genentech has announced positive results from its primary analysis of an ongoing study assessing safety and efficacy of Evrysdi in babies with pre-symptomatic spinal muscular atrophy.

Despite the number of new medications produced to treat a range of neurologic diseases, limited utilization is driven by relatively high cost and similar efficacy to less expensive drugs, according to a study published in Neurology.

Treatment with nusinersen led to continued and improved motor function up to 5 years after initial dose in infants with spinal muscular atrophy, according to a poster presented at the International Scientific Congress on Spinal Muscular Atrophy.

Daily treatment with risdiplam over 24 months resulted in improved motor function and achievement of developmental motor milestones in infants with type 1 spinal muscular atrophy, according to a study published in The Lancet Neurology.

Genentech announced that the FDA approved a label extension for Evrysdi to include infants younger than 2 months with spinal muscular atrophy.

The FDA has granted priority review to PTC Therapeutics Inc. for a supplemental new drug application regarding Evrysdi, according to a company release.

Biogen announced it intends to initiate a global phase 3b study to investigate clinical outcomes and safety of a higher dose of Spinraza in patients with later onset spinal muscular atrophy previously treated with Evrysdi.