The two talk about the advances in medicine and treatments for SMA, in particular, the success seen in children with type 1 SMA who have undergone the gene therapy AVXS-101. The clinical trials have shown SMA infants who would normally be unable to breathe unaided at eight months are able to crawl like normal babies following the treatment. Peter and Michaela talk about the possibility of the treatment being passed by the FDA and applied to other similar diseases. Find out more about the clinical trial for AVXS-101 here.
SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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