News

Delayed Spinraza (nusinersen) treatment due to the COVID-19 pandemic did not directly result in a worsening of symptoms in children with spinal muscular atrophy (SMA), a small study suggests. Any worse motor function that occurred during this period was attributed to a lack of response to the therapy…

Genetic analysis of dried saliva spots identified people with spinal muscular atrophy (SMA), a study demonstrated and possibly for the first time. The scientists noted that this noninvasive and low-cost, alternate method of genetic screening may be suitable for programs held in schools or workplaces without the need for…

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…

The COVID-19 pandemic led to delays in treatment with Spinraza (nusinersen) for many children with spinal muscular atrophy (SMA). However, these delays seem to have a lower impact on children’s functional skills when compared with family support, according to a small study in Italy. The study, “…

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…

The blood levels of neurofilaments — a marker of nerve cell damage — and the results of a nerve-muscle test called compound muscle action potential (CMAP) may serve as a biomarker of disease onset and treatment response in infants and children with spinal muscular atrophy (SMA) up to 3…

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…

A University of Michigan (U-M) senior with spinal muscular atrophy (SMA) has won the school’s James T. Neubacher Award for his efforts to help make the campus — and everywhere else — more accessible to people with physical disabilities. Vincent Pinti, a political science major minoring in Spanish,…

The U.S. Food and Drug Administration (FDA) has added acute liver failure to the list of safety concerns with use of Zolgensma (onasemnogene abeparvovec-xioi) in children with spinal muscular atrophy (SMA). The therapy’s label has been updated to contain a boxed warning highlighting the risk of higher-than-normal…

Motor function and stabilizing or improving breathing — along with oral administration — are the factors that adult patients and caregivers of children with spinal muscular atrophy (SMA) type 2 or 3 who cannot walk value most when deciding on available treatments, a U.K. survey study shows. Avoiding…