2don MSN
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 ...
The Chosun Ilbo on MSN
British boy walks after world's costliest gene therapy
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, ...
After five years, Nido Biosciences has reached the end of the road, closing its doors after its lead neurological disease ...
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 ...
Join the community workshop in Thiruvananthapuram to support families affected by Spinal Muscular Atrophy and learn about the condition.
—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 ...
4don MSN
Abu Dhabi leads world in breakthrough gene replacement therapy 'ITVISMA' for spinal muscular atrophy
Abu Dhabi marks landmark achievement in becoming the first in the world to deliver ITVISMA (onasemnogene abeparvovec) for the ...
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.
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