News

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…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

Lung function and upper limb abilities worsened over a two-year period in young people with spinal muscular atrophy (SMA) type 3 who were unable to walk (non-ambulatory), a U.K. study has found. At the time of the study, the approved disease-modifying therapy Spinraza (nusinersen) was unavailable to SMA…

[Editor’s note: An earlier version of this story did not specify in its opening statement that Spinraza’s use in this proposed trial would follow Evrysdi’s use.] Biogen plans to launch a Phase 3b trial into the safety and efficacy of a higher dose of Spinraza (nusinersen) in children,…

Blood levels of neurofilament light chain (NfL) — a marker of nerve cell damage — in children and adolescents with spinal muscular atrophy (SMA) are significantly higher than those of age-matched healthy peers and associate with disease severity, a study found. NfL blood levels also reliably reflect levels in…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

A naturally occurring form of the androgen receptor protein — which scientists have named AR45 because of its size — may be useful for treating spinal and bulbar muscular atrophy (SBMA), a rare form of adult-onset spinal muscular atrophy, a new study suggests.

Cure SMA and Cytokinetics have extended a partnership helping to heighten spinal muscular atrophy (SMA) education, awareness and fundraising, and t0 direct public policy in addressing community needs. To that end, the biopharmaceutical company will again be a National Gold Sponsor of some upcoming Cure SMA efforts…