onasemnogene abeparvovec Zolgensma
Selected indexed studies
- Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma). (Cureus, 2023) [PMID:37065340]
- 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. (Lancet Neurol, 2021) [PMID:33743238]
- Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy. (CNS Drugs, 2022) [PMID:35960489]
_Worker-drafted node — pending editorial review._
Connections
onasemnogene abeparvovec Zolgensma is a side effect of
Sources
- Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma). (2023) pubmed
- 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. (2021) pubmed
- Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy. (2022) pubmed
- Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. (2022) pubmed
- Onasemnogene Abeparvovec. (2012) pubmed
- Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. (2021) pubmed
- PMID:36126155 (2021) pubmed
- Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue. (2021) pubmed
- Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis and Treatment. (2023) pubmed
- An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies. (2024) pubmed