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 ...

Ocugen ( OCGN -6.48%) Q3 2024 Earnings Call Nov 08, 2024, 8:30 a.m. ET Good morning and welcome to Ocugen's third quarter 2024 financial results and business update. [Operator instructions] I will now ...

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.

Mapping cell types in MS lesions reveals unique tissue niches and interactions, offering insights into the molecular drivers of multiple sclerosis progression.

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

Subsequent to quarter-end, closed $30 million in debt financingOCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) on track to ...