The health activists argue that the government is empowered with effective law and policy tools and therefore should ...

Columnist Helen Baldwin gains a different perspective following a crushing diagnosis, but glasses weren't what helped her to see clearly.

The mutation that causes Spinal Muscular Atrophy (SMA) can be detected in a prenatal screen. But a parent's decision to test for this terrible disease is not as easy as you would think.

Mesenteric (or intestinal) artery disease is a condition that develops when the arteries in the abdomen that supply the intestines become narrowed, or blocked, by an accumulation of a fatty substance ...

A woman with SMA type 3 became pregnant while on Spinraza treatment and gave birth to a healthy child, per a case report.

Five more rare diseases have been identified in the country, making the total number of rare diseases reach 456, according to the Rare Disease Foundation.

Our current AAV9 portfolio includes gene therapy candidates for SMA, Rett syndrome, SOD1 amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia, with several other AAV9-based candidates in ...