News

Among children with neuromuscular disorders, those with spinal muscular atrophy type 1 (SMA 1) appear to be more vulnerable to more severe COVID-19 symptoms, a small study suggests. However, the course of disease in children with neuromuscular disorders overall may not be as…

Four measures of motor ability — including one of continuous upper limb activity that can be done at the home — reliably detect significant functional changes over one or two years in people with spinal muscular atrophy (SMA) types 2 and 3, according to a natural history…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a well-deserved…

A free resource booklet, published by Cure SMA, may help people with spinal muscular atrophy (SMA) to better understand the possible risks and benefits of combining different treatments for the disease. The booklet, titled “Scientific Considerations for Drug Combinations,” is available online. The past several years…

Efficacy and safety results from the TOPAZ Phase 2 trial evaluating apitegromab, a  muscle-directed therapy for spinal muscular atrophy (SMA) being developed by Scholar Rock, are expected by June, the company announced. “2020 was a transformative year for Scholar Rock with … positive interim data from the TOPAZ…

The COVID-19 pandemic has forced many activities to be online only, but in the case of this year’s Team Cure SMA endurance race series, the limitation means more people can join. Register here for the May 23 virtual endurance event, in which participants can bike, run, or walk to raise…

The first patient has been dosed in a Phase 4 trial exploring the safety and potential benefits of Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) who failed to respond adequately to Zolgensma. The two-year study, called RESPOND (NCT04488133), was launched last year by…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for a…

Cure SMA has developed a new three-part webinar series that delves into health and wellness topics pertinent to the spinal muscular atrophy (SMA) community. The three monthly installments of the Wellness Webinar Series will teach participants about a variety of strategies and tools that can be used…

After finding no safety concerns in Part A of the DEVOTE trial, which is testing higher doses of Spinraza (nusinersen) among people of all ages with spinal muscular atrophy (SMA), investigators are now screening patients for the study’s pivotal Part B, according to Biogen, the therapy’s maker. Part B,…