Aviana McElwee from Darwin, Australia, was just a few months old when her doctor realized she was having problems reaching milestones. Tests later confirmed that she had spinal muscular atrophy (SMA). According to a report from abc.net.au, Aviana is the ninth baby in the world to receive the drug nusinersen (Spinraza) for free under the Expanded Access Program (EAP) while local hospitals and health authorities are waiting to be granted approvals for the drug.
In just two months, Aviana’s parents and doctor have seen a marked improvement in her strength, mirroring what many of the babies on the clinical trial experienced with the drug. They were able to meet unexpected milestones such as crawling, standing and even walking.
Nusinersen has yet to be approved in Australia, but the SMA community there is watching with interest.
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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