Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, intrathecal onasemnogene abeparvovec (OAV101 IT) in treating individuals with spinal ...
Spinal muscular atrophy (SMA) 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 ...
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What is spinal muscular atrophy?
Former Little Mix star Jesy Nelson has shared the devastating news that her twin daughters, Ocean Jade and Story Monroe Nelson-Foster, have been diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a ...
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