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1 Indications And Usage Zolgensma (onasemnogene Abeparvovec-Xioi) Is An Adeno-Associated Virus Vector-Based Gene Therapy Indicated For The Treatment Of Pediatric Patients Less Than 2 Years Of Age With Spinal Muscular Atrophy (sma) With Bi-Allelic Mutations In The Survival Motor Neuron 1 (smn1) Gene. Limitation Of Use • The Safety And Effectiveness Of Repeat Administration Of Zolgensma Have Not Been Evaluated [ See Adverse Reactions ( 6.2 ) ] . • The Use Of Zolgensma In Patients With Advanced Sma (e.g., Complete Paralysis Of Limbs, Permanent Ventilator-Dependence) Has Not Been Evaluated [ See Clinical Studies ( 14 ) ]. Zolgensma (onasemnogene Abeparvovec-Xioi) Is An Adeno-Associated Virus Vector-Based Gene Therapy Indicated For The Treatment Of Pediatric Patients Less Than 2 Years Of Age With Spinal Muscular Atrophy (sma) With Bi‑allelic Mutations In The Survival Motor Neuron 1 ( Smn1) Gene. ( 1 ) Limitation Of Use: • The Safety And Effectiveness Of Repeat Administration Of Zolgensma Have Not Been Evaluated. ( 1 , 6.2 ) • The Use Of Zolgensma In Patients With Advanced Sma (e.g., Complete Paralysis Of Limbs, Permanent Ventilator Dependence) Has Not Been Evaluated. ( 1 , 14 )
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