Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio
Author:
Publisher
Elsevier BV
Subject
Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health
Reference16 articles.
1. Natural history of infantile-onset spinal muscular atrophy;Kolb;Ann Neurol,2017
2. Longitudinal natural history of type I spinal muscular atrophy: a critical review;Mercuri;Orphanet J Rare Dis,2020
3. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial;Day;Lancet Neurol,2021
4. Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy;Mendell;JAMA Neurol,2021
5. Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series;Chand;J Pediatr,2021
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