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

Making a situation accessible is more about mindset than things, says columnist. That's why attitude is important.

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, ...

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 ...

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 ...