Scholar Rock is once again seeking approval in Europe for its muscle-strengthening drug for SMA, already launching in the U.S. as Isembyld.

The arrival of a new night weekend nurse has given columnist Ari Anderson reason to believe that he's now on the path of ...

There are several treatment options available for spinal muscular atrophy (SMA), a rare genetic condition characterized by progressive muscle weakness and wasting. The disease mainly affects motor ...

England has launched SMA newborn screening in the West Midlands, marking the first step toward a nationwide NHS rollout.

Columnist Sherry Toh may be waiting to try Isembyld, but the new SMA treatment should still be made accessible quickly, she ...

Columnist Connie Chandler recalls a trip she took to the Rocky Mountains, and how the challenges were a call to go further up ...

A compound in some fruits may be a treatment candidate for SMA, according to a study that explored the effects of autophagy ...

For many people living with rare neuromuscular diseases, the most meaningful part of a recent Chicago gathering was not just the medical updates — it was finding a community that understood their ...

Children with SMA types 2 and 3 treated with two approved meds also saw sleep-related breathing problems ease over time, a ...

People with SMA, caregivers, and healthcare professionals in Europe identified 10 research priorities that could have the ...

New research reveals that the front and back parts of the brain develop independently from completely separate progenitor cells. This discovery helps explain how SMA affects the hindbrain and allows ...