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Additionally, Risdiplam (Evrysdi) is an orally administered small molecule that promotes SMN2 exon 7 inclusion, enhancing SMN protein synthesis 6

Reporting and communicating results 7.1 Reporting results to CHRDs/CHISs Screening laboratories and CHRDs/CHISs should use the national status codes and subcodes to record the outcomes of NBS screening

It plays a pivotal role in energy production by transporting fatty acids into the mitochondria, where they can be burned for energy