LONG-TERM FOLLOW-UP STUDY OF PATIENTS WITH SPINAL MUSCULAR ATROPHY RECEIVING RISDIPLAM TREATMENT

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LONG-TERM FOLLOW-UP STUDY OF PATIENTS WITH SPINAL MUSCULAR ATROPHY RECEIVING RISDIPLAM TREATMENT

Brief description of study

This study will evaluate the long-term safety and effectiveness of risdiplam (Evrysdi?), prescribed on the basis of the U.S. Package Insert (USPI) and clinician judgement, in adult and pediatric spinal muscular atrophy (SMA) patients.

Clinical Study Identifier: s22-00184

Principal Investigator: Mary-Lynn Y Chu.

Other Investigator: Patrick Kwon.

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