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Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.

Ann Clin Transl Neurol · 2023 · PMC10646995 · PMID 37691296

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a clear trendno p-value reported
1 , 2 , 3 , 4 , 5 Despite a lack of clinical trial and real‐world data to demonstrate the clinical benefit (or risk) associated with the administration of more than one SMN ‐targeted DMT in combination or in sequence, a clear trend exists for the use of more than one DMT in specific circumstances (e.g., initiating bridge treatment with nusinersen or risdiplam while awaiting decline of anti‐AAV9 [adeno‐associated virus serotype 9] antibody titers to a concentration that permits onasemnogene abeparvovec administration or physic

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