Post surgery the patient had to undergo physiotherapy and his lifelong wish to sit straight without falling has come true. He now has a 'strong and straight' spine. This is one of the finest ...

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

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, ...

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

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

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

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

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

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

Over a series of conferences during the third quarter 2024, Ocugen had the opportunity to provide an update on its three ...