Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical trials supporting the therapy’s approval. These were the findings of an analysis of safety data from the U.S.

A second-generation investigational gene therapy for spinal muscular atrophy (SMA) showed greater safety and efficacy than an earlier version, and it may be more effective than the currently approved gene therapy for SMA, a study in a disease mouse model reported. The study was conducted by scientists with CANbridge…

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

As part of ongoing efforts to have all newborns in the U.K. screened for spinal muscular atrophy (SMA), Muscular Dystrophy UK (MDUK) recently met with an ad hoc Scottish group to hear from parents and clinicians about life with SMA and the benefits of early diagnoses. The meeting, hosted…

The availability of early genetic testing and approval of several spinal muscular atrophy (SMA) treatments has dramatically changed disease outcomes, while introducing ethical issues in terms of screening, predicting SMA severity, and patient access to therapy. As such, appropriate information and support should be given to parents and patients…

Playing video games can be a challenge for people with spinal muscular atrophy (SMA). Multiplayer games may pit them against those who are able-bodied, action role-playing games force them to do multiple motions on a controller with one hand, and fighting games reward players for quick-twitch muscle moves. Painted…

Treatment with Evrysdi (risdiplam) continues to be safe and well tolerated in people with spinal muscular atrophy (SMA) who have previously received other therapies for SMA, according to one-year data from the JEWELFISH clinical trial. Results also suggest that motor function has been stable in JEWELFISH participants after a…

Unlike in Australia, the U.S., and even several EU countries, there have been no programs for newborn screening for spinal muscular atrophy (SMA) in the United Kingdom — despite compelling evidence that an early diagnosis can improve patient outcomes. Now, Oxford University in the U.K. has launched a pilot study…

Babies born in the Canadian province of Saskatchewan will now be tested for spinal muscular atrophy (SMA) as part of routine newborn screening. SMA is one of four medical conditions that have been added to the screening program administered to newly born infants by the Saskatchewan Health Authority. “Our…

Children, teenagers, and adults with later-onset spinal muscular atrophy (SMA), previously treated with Evrysdi (risdiplam), can now enroll in a new Phase 3b clinical trial to determine the efficacy of a higher dose of Spinraza (nusinersen). Biogen, the company that markets Spinraza, announced the enrollment of…