A 5-year-old boy who was born with a rare genetic condition is now able to walk by himself, his mother has said, after ...

A 5-year-old British boy suffering from the rare disease spinal muscular atrophy (SMA) has miraculously begun to walk four years after receiving the "world’s most expensive" gene therapy. On the 31st, ...

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

A multidisciplinary panel of experts discusses the clinical burden of spinal muscular atrophy on patients, families, and caregivers and considers the importance of patient education and advocacy.

Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...

—The slow progression of SMA in adults and older children complicates evaluation of treatment response to newer therapies. Longitudinal quantitative MRI (qMRI) may give clinicians a better handle on ...

Join the community workshop in Thiruvananthapuram to support families affected by Spinal Muscular Atrophy and learn about the condition.

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...

After five years, Nido Biosciences has reached the end of the road, closing its doors after its lead neurological disease ...