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 ...

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.

Akari Therapeutics, Plc (Nasdaq: AKTX), an innovative biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, announces shareholder approval in connection with ...

OCU410 is being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 2-3 million people in the U.S. & EU. Current ...

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

The company is developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including inherited retinal diseases ("IRDs"), such as RP, LCA, ...

Bethel University is hosting a charity basketball game Saturday to raise funds for a local teenage girl with spinal muscular atrophy, or S.M.A.