The European Commission (EC) has approved a tablet formulation of spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam). “The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche,…
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A quantitative MRI scan, which shows detailed images of muscles, can be used to track changes seen with Spinraza (nusinersen) treatment in children with spinal muscular atrophy (SMA), according to a new study from China. SMA children started on the approved therapy at a younger age also were found to…
Offering children with spinal muscular atrophy (SMA) type 1 the opportunity to move around on their own using motorized, ride-on cars may boost their confidence, help them connect with others, and make them feel more included, a small study found. “Powered mobility not only facilitates movement but also promotes…
After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed improved motor function and more independence in tasks involving their arms and hands, a study from China has found. These motor function improvements were linked to reduced symptoms of anxiety…
Treatment with AJ201 was well tolerated and showed signs of improving physical activity in a clinical trial involving people with spinal and bulbar muscular atrophy (SBMA), according to new data announced by developer Annji Pharmaceutical. “I am greatly encouraged by the positive clinical outcomes observed after a relatively…
Injecting medicines into the amniotic fluid — the liquid that surrounds a developing fetus during pregnancy — may be a viable strategy for treating spinal muscular atrophy (SMA) before birth, according to a new study done in animal models. “These data demonstrated that [injecting therapies into amniotic fluid] holds…
Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type 3 and to be safe in non-human primates, supporting its move into clinical testing. Genecradle Therapeutics is sponsoring ongoing Phase 1/2 clinical trials in SMA type 1 (NCT05824169),…
Blood creatinine-to-cystatin-c ratio, an emerging indicator of muscle mass and strength, may be a promising biomarker of therapeutic response in adults with spinal muscular atrophy (SMA), a study in China shows. Increases in the ratio were significantly associated with gains in motor function, including improved motor skills, hand and…
Training with a wearable robotic device called HAL, for hybrid assistive limb, improved walking ability for people with spinal muscular atrophy (SMA) who were also receiving treatment with Spinraza (nusinersen), according to a study in Japan. “[These] results may provide sufficient data to inform clinical decision-making about the…
Richard Finkel, MD, a pediatric neurologist at St. Jude Children’s Research Hospital, was included on a list of 100 individuals having a positive influence on global health in 2025, in recognition of his work treating childhood neuromuscular disorders such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Finkel…
