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 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.

—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 ...

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

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

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

The loss of muscle mass with increased fat mass in patients with SMA suggests a dietary imbalance between energy intake and expenditure. Dietary modifications have shown some promise in managing SMA, ...

Abu Dhabi marks landmark achievement in becoming the first in the world to deliver ITVISMA (onasemnogene abeparvovec) for the ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

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 ...

India makes many of the world’s drugs, but treatments for rare diseases like spinal muscular atrophy are imported and prohibitively costly. In desperation, parents are raising funds on social media.