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

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

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

In May 1997, my husband, Randy, and I awaited the arrival of our third baby. The ultrasound indicated we were having a boy. We named him Jeffrey. Years before, I’d taught at Brockman School, a ...

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