News

About one year of treatment with Spinraza (nusinersen) gradually and significantly improves motor function in adults with spinal muscular atrophy (SMA) type 3 — especially those unable to walk without assistance, according to a large and real-life study in Italy. Adults with type 2 disease, in…

Evrysdi (risdiplam), Roche’s oral therapy for spinal muscular atrophy (SMA), will be available to select patients in the U.K. before its potential regulatory approval through an Early Access to Medicines Scheme (EAMS). Eligible patients include those with type 1 and 2 disease, ages…

Control Bionics has announced the launch of the GridPad Trilogy, an augmentative and alternative communication (AAC) device intended to aid people living with a range of conditions that affect movement and speech, such as spinal muscular atrophy…

To help children with spinal muscular atrophy (SMA) gain mobility and independence, Electric Bike Technologies (EBT) is donating 10 Liberty Trikes — and plans to double that gift next year. The Liberty Trike is a folding and active-mobility tricycle that can be used by children with SMA…

In previous articles, we have discussed different techniques for manual stretching to improve functional mobility and range of motion for Michael, my patient with SMA. We have discussed the importance of maintaining and increasing range of motion in joints such as ankles, knees, hips, elbow, hand, and cervical spine. These…

Two years after acquiring the gene therapy company AveXis, Novartis has renamed it Novartis Gene Therapies to underscore the potential value of developing such treatments for genetic diseases. This decision was based partly on the success of Zolgensma, originally developed by AveXis, as…

England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), according to a press release from SMA UK. NICE had previously planned to conduct evaluations of clinical trial and cost-effectiveness data,…

Treatment combinations, including future muscle-targeted therapies, are part of what likely lies ahead for spinal muscular atrophy (SMA), according to experts in the SMA community. In separate interviews with SMA News Today, Richard Finkel and Laurent…

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. Baby’s First Test is a program of Expecting Health, an organization focused on pregnancy and newborn health.

SMA Europe has launched an alliance that is demanding that, by 2025, newborn screening programs in Europe include a test for spinal muscular atrophy (SMA). The new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance) was launched as a final initiative to mark…