Melatonin and pioglitazone, two approved drugs that act on metabolism, improved motor function and prolonged survival in ...
SMA treatment Evrysdi partly normalized changes in molecules tied to inflammation and cellular stress in adults with ...
When it comes to disability inclusion, language is powerful, but it needs to be supported by action, argues columnist Sherry ...
Columnist Connie Chandler shares three things in her life with SMA that she thinks someone who might want to spend time with ...
It started with what I had assumed was an ordinary breakout. I’d been stressed from moving for the first time in my life, and had punted on my typically zealous hygiene because, well, moving. You ...
Treatment with Evrysdi (risdiplam) was associated with stable overall motor function and improved upper limb function in a small Chinese study of 15 people with spinal muscular atrophy (SMA). Upper ...
This Halloween, an 11-year-old living with SMA named Dash will be trick-or-treating in a custom wheelchair costume inspired ...
Dallas, keynote speaker Lily Sander discusses belonging, resilience, and the power of community and connection.
In getting ready for the day, columnist Connie Chandler's caregiver helps her look good on the outside, but Connie's attitude ...
Scholar Rock has resubmitted an application in the European Union seeking regulatory approval of its muscle-strengthening drug apitegromab. The infusion therapy won FDA approval last month and is ...
England launched a new NHS newborn screening initiative to test babies for SMA, starting regionally in the West Midlands. SMA is a rare genetic condition causing progressive muscle weakness due to the ...