Risk-benefit profile of onasemnogene abeparvovec in older and heavier children with spinal muscular atrophy type 1

Author:

Finnegan Rebecca,Manzur Adnan,Munot Pinki,Dhawan Anil,Murugan Archana,Majumdar Anirban,Wraige Elizabeth,Gowda Vasantha,Vanegas Maria,Main Marion,O'Reilly Emer,Baranello Giovanni,Muntoni Francesco,Scoto Mariacristina

Publisher

Elsevier BV

Reference12 articles.

1. Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study;Weiß;Lancet Child Adolesc Health,2022

2. National Institute for Health and Care Excellence: risdiplam for treating spinal muscular atrophy. December 2023. Risdiplam for treating spinal muscular atrophy (nice.org.uk)

3. National Institute for Health and Care Excellence: nusinersen for treating spinal muscular atrophy. July 2019. Nusinersen for treating spinal muscular atrophy (nice.org.uk)

4. National Institute for Health and Care Excellence: onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy. April 2023. 1 Recommendations | Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy | Guidance | NICE

5. Onasemnogene abeparvovec for the treatment of spinal muscular atrophy;McMillan;Expert Opin Biol Ther,2022

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