The SBMA therapy AJ201 showed good safety, hints of efficacy, and improved physical activity for this SMA type in a trial, ...

Injecting medicines into amniotic fluid may be a viable strategy for treating SMA before birth, according to a new study in ...

“Umm, bunk beds?” My mom and I stopped in our tracks, backpacks and pillows in tow, peering into the room where we were supposed to sleep during a fun and relaxing weekend away. Needless to say, it ...

A wearable robotic device called HAL improved walking ability for SMA patients who also received Spinraza (nusinersen), a ...

Blood creatinine-to-cystatin-c ratio, an indicator of muscle mass and strength, may be a treatment response biomarker of SMA, ...

Richard Finkel, MD, a pediatric neurologist at St. Jude Children’s Research Hospital, was included in the Time100 Health list ...

SMA gene therapy GC101 was found to rescue disease manifestations in mice and to be safe in non-human primates.

Michela Luciano is a Science Writer at BioNews. Originally from the picturesque mountain town of L’Aquila, Italy, Michela ...

Columnist Connie Chandler explains why it's important to give kids the chance to ask questions about disability — and answer them honestly.