Jesy Nelson, Spinal Muscular Atrophy
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Boy, 5, Born with Rare Genetic Disorder Walks by Himself After Receiving World’s Most Expensive Drug
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 receiving the world's most expensive drug, which costs around $2.4 million.
The Independent on MSN
What is spinal muscular atrophy?
Spinal muscular atrophy explained after Jesy Nelson reveals her twins’ diagnosis - The singer shared an emotional update about her twins’ health on Sunday and that ‘time is of the essence with this di
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 lead to severe physical disability, paralysis and life ...
Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no signs at all two and a half years after birth.
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 investigational gene therapy for young people with ...