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

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

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.

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.

—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 ...

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 ...

Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades. 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 ...

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, ...

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.