News

In a letter to a U.S. Department of Transportation Advisory Committee, Cure SMA called for better wheelchair handling and storage for people with spinal muscular atrophy (SMA) who travel by air. The patient advocacy organization shared its perspective on the challenges of airplane travel — from booking tickets to…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

The Canadian Agency for Drugs and Technologies in Health (CADTH) will not recommend reimbursement for Zolgensma in children with spinal muscular atrophy (SMA) who are more than 6 months old, citing insufficient evidence of efficacy and safety in older patients. “Other countries are providing guidance and funding based…

Scholar Rock plans to soon initiate a Phase 3 clinical trial of apitegromab, its investigational muscle-directed treatment for spinal muscular atrophy (SMA), in type 2 and 3 patients who are unable to walk. “We anticipate initiating a Phase 3 trial to evaluate apitegromab in patients with non-ambulatory Type…

In infants with spinal muscular atrophy (SMA) type 1, lower blood levels of phosphorylated neurofilament heavy chain (pNF-H) — a marker of nerve cell damage — before starting treatment with Spinraza (nusinersen) are significantly associated with greater motor gains over time. These findings, based on data from the Phase…

Bionews Insights is launching a new survey, in collaboration with SMA News Today, with the goal of understanding how different aspects of this disease affect an individual’s quality of life. The survey, which is expected to take about 15 minutes to complete, opens with WHOQOL-100, a set of questions developed…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA) have retained the ability to swallow, and most have been able to stand and walk within developmentally normal windows. That’s according to new data from the RAINBOWFISH clinical trial presented…

Biogen is continuing to actively recruit patients for its Phase 4 RESPOND trial, which is evaluating the benefits of Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma. Participants are now being enrolled at 10 sites in the U.S. and one in Italy,…

Children with spinal muscular atrophy (SMA) type 2 or 3 in China have lower bone mineral density — putting them at increased risk for bone fractures — compared with healthy children of the same age and sex, a study has found. Furthermore, SMA type 2 children had significantly…