News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. FDA grants priority review to risdiplam for the treatment of SMA

    The United States’ Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for risdiplam (RG7916) for the treatment of SMA. The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA. […]

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