Public health groups are challenging Roche's attempt to block a generic version of its expensive Spinal Muscular Atrophy drug ...

Gene therapy for Spinal Muscular Atrophy (SMA) is prohibitively expensive in India, with the life-saving drug Zolgensma ...

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 ...

This has created a desire for people to maintain that relationship for as long as possible and to ensure that the quality of ...

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

PulseSight plans to submit a phase I clinical trial authorization (CTA) by the end the year, to be closely followed by a phase II proof-of-concept to demonstrate the efficacy and the safety of its ...

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

Despite diagnostic and therapeutic advances, mortality and the multi-systemic health impact of SMA continue to be experienced ...

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.