Australian Baby Becomes Ninth SMA Patient to Receive Nursinersen
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.
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