We must understand that while rare diseases, by definition, affect a small portion of the population, they collectively ...

Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...

Researchers have uncovered a mechanism that may trigger ALS’s earliest stages, identifying proteins that mislocalize, causing ...

Approximately 5,000 people in the U.S. develop amyotrophic lateral sclerosis (ALS) each year. On average, they survive for ...

A rare spinal surgery in Thiruvananthapuram, India, has transformed the life of 23-year-old Mohamad Raishan Ahmed from the ...

A research team at the University of California has uncovered a link between sleep quality in midlife and brain atrophy, a ...

A 36-week treatment regimen of pegcetacoplan reduces geographic atrophy growth rate while demonstrating a favorable safety profile.

Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, ...

Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.

"With the support of our shareholders at the General Meeting, we are targeting an official close of the transaction on ...

A 57-year-old man suffering from debilitating pain, numbness, and difficulty walking found relief through immunotherapy, ...

Williams appears gaunt, which experts say might indicate nutritional deficiencies A photo of a NASA astronaut putting ...