News

Treatment with AJ201 shows positive signs in SBMA clinical trial

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…

SMA gene therapy GC101 found safe, effective in animals

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),…

Wearable robotic device may boost walking ability for SMA patients

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…

St. Jude pediatric neurologist included on Time100 health list

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…

SMA Europe’s inaugural gathering to discuss medical emergencies

SMA Europe will host its 1st International Clinical Care Symposium on SMA and focus on the biological causes of spinal muscular atrophy (SMA) and how to prevent and manage the symptoms that may lead to life-threatening emergencies. Titled, “Medical Emergencies in SMA: pathophysiology, prevention, and response,” the…

High-carbohydrate diet may help normalize SMA metabolism

A study found that people with spinal muscular atrophy (SMA) have metabolic alterations due to impairments in how their bodies use fatty acids for energy. The results suggested a carbohydrate-rich diet may help normalize metabolism in SMA patients. The study, “Alterations of Fat and Ketone Body Metabolism…