Adults with spinal muscular atrophy (SMA) type 2 have lower intelligence quotient (IQ) scores on assessments for working memory and perceptual reasoning compared with the general population, according to data from a new study. The study, “Cognitive Impairment in Adult Patients with 5q-Associated Spinal Muscular Atrophy,” was published in…
A “significant” economic burden is associated with spinal muscular atrophy (SMA), although differences in healthcare costs and resource use are evident among patients by SMA type, a large review study found. Not surprisingly, the greater burden is among patients with SMA type 1, one of this disease’s most…
Not having Spinraza (nusinersen) therapy as prescribed — called treatment non-adherence by clinicians — increased the frequency of co-existing diseases, required greater use of healthcare resources, and raised overall costs for patients with spinal muscular atrophy (SMA) in the U.S. and their families, retrospective research into claims data for…
Scholar Rock is on track to initiate a pivotal Phase 3 trial of apitegromab in non-ambulatory patients with type 2 and type 3 spinal muscular atrophy (SMA) by the end of 2021, according to a company press release. The planned randomized, double-blind, and placebo-controlled trial will focus on people…
Novartis Pharmaceuticals Canada is lauding the province of Quebec for its decision to provide public reimbursement for Zolgensma (onasemnogene abeparvovec), an approved gene therapy for spinal muscular atrophy (SMA) in children. Zolgensma is the first gene therapy to receive formal public reimbursement in Canada. The current recommendation…
