The film explains the genetic defects which lead to SMA, the faulty genes involved and how nusinersen enables patients to make more of the vital SMN protein.
Nusinersen was approved by the Food and Drug Administration (FDA) in December 2016 to treat babies, children, and adults with the disease. Clinical trials showed promising results with many babies with type 1 SMA reaching milestones such as rolling, crawling, head control, standing and walking–milestones usually not met by children with the worst type of the disease. Find out more about the FDA’s approval of nusinersen here.
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