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Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy.
Blair HA. Blair HA. CNS Drugs. 2022 Sep;36(9):995-1005. doi: 10.1007/s40263-022-00941-1. Epub 2022 Aug 12. CNS Drugs. 2022. PMID: 35960489 Review.
Onasemnogene abeparvovec (Zolgensma()) is a gene therapy approved for the treatment of spinal muscular atrophy (SMA). ...Onasemnogene abeparvovec was generally well tolerated. Hepatotoxicity is a known risk that can generally be mitigated with prophyla …
Onasemnogene abeparvovec (Zolgensma()) is a gene therapy approved for the treatment of spinal muscular atrophy (SMA). ...On …
Onasemnogene Abeparvovec.
[No authors listed] [No authors listed] 2020 Aug 20. LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012–. 2020 Aug 20. LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012–. PMID: 33242238 Free Books & Documents. Review.
Onasemnogene abeparvovec is a unique gene therapy agent used to correct the abnormal gene responsible for spinal muscular atrophy type 1, a rare progressive neuromuscular disorder that typically leads to disability and death within the first two years of life. On …
Onasemnogene abeparvovec is a unique gene therapy agent used to correct the abnormal gene responsible for spinal muscular atro …
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 JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Peña LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Day JW, et al. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17. Lancet Neurol. 2021. PMID: 33743238 Clinical Trial.
All patients who received onasemnogene abeparvovec had at least one adverse event (most common was pyrexia). ...INTERPRETATION: Results from this multicentre trial build on findings from the phase 1 START study by showing safety and efficacy of commercial grade o …
All patients who received onasemnogene abeparvovec had at least one adverse event (most common was pyrexia). ...INTERPRETATION …
Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data.
Erdos J, Wild C. Erdos J, et al. Eur J Paediatr Neurol. 2022 Jul;39:1-10. doi: 10.1016/j.ejpn.2022.04.006. Epub 2022 Apr 30. Eur J Paediatr Neurol. 2022. PMID: 35533607 Free article. Review.
RESULTS: Five hundred forty-six records were identified in the literature search and 22 studies (in 26 publications) were included in the analysis. Nusinersen, onasemnogene abeparvovec and combination therapies improved motor endpoints in SMA type 1 patients. ...
RESULTS: Five hundred forty-six records were identified in the literature search and 22 studies (in 26 publications) were included in the an …
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis.
Pascual-Morena C, Cavero-Redondo I, Lucerón-Lucas-Torres M, Martínez-García I, Rodríguez-Gutiérrez E, Martínez-Vizcaíno V. Pascual-Morena C, et al. Hum Gene Ther. 2023 Feb;34(3-4):129-138. doi: 10.1089/hum.2022.161. Hum Gene Ther. 2023. PMID: 36136906 Review.
One of the latest approved therapies for spinal muscular atrophy (SMA) is onasemnogene abeparvovec, which transduces motor neurons with the survival of motor neuron gene. The aim of this meta-analysis was to estimate the effect of onasemnogene abeparvovec …
One of the latest approved therapies for spinal muscular atrophy (SMA) is onasemnogene abeparvovec, which transduces motor neu …
Onasemnogene Abeparvovec: First Global Approval.
Hoy SM. Hoy SM. Drugs. 2019 Jul;79(11):1255-1262. doi: 10.1007/s40265-019-01162-5. Drugs. 2019. PMID: 31270752 Review.
Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA()) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cell …
Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA()) is an adeno-associated …
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM, Chiriboga CA, Iannaccone ST, Krueger JM, Parsons JA, Shieh PB, Kavanagh S, Wigderson M, Tauscher-Wisniewski S, McGill BE, Macek TA. Strauss KA, et al. Nat Med. 2022 Jul;28(7):1390-1397. doi: 10.1038/s41591-022-01867-3. Epub 2022 Jun 17. Nat Med. 2022. PMID: 35715567 Free PMC article. Clinical Trial.
SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated within six postnatal weeks. ...No serious adverse events were consi …
SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvov …
Onasemnogene abeparvovec for spinal muscular atrophy.
[No authors listed] [No authors listed] Aust Prescr. 2022 Aug;45(4):140-141. doi: 10.18773/austprescr.2022.044. Epub 2022 Jul 7. Aust Prescr. 2022. PMID: 36110172 Free PMC article. Review. No abstract available.
Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma).
Ogbonmide T, Rathore R, Rangrej SB, Hutchinson S, Lewis M, Ojilere S, Carvalho V, Kelly I. Ogbonmide T, et al. Cureus. 2023 Mar 15;15(3):e36197. doi: 10.7759/cureus.36197. eCollection 2023 Mar. Cureus. 2023. PMID: 37065340 Free PMC article. Review.
The objective of the study is to review the safety and efficacy of a novel gene therapy, onasemnogene abeparvovec (Zolgensma), for SMA and assess current challenges for gene therapy. ...Therefore, the combined safety, cost, and effectiveness of onasemnogene …
The objective of the study is to review the safety and efficacy of a novel gene therapy, onasemnogene abeparvovec (Zolgensma), …
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.
Thomsen G, Burghes AHM, Hsieh C, Do J, Chu BTT, Perry S, Barkho B, Kaufmann P, Sproule DM, Feltner DE, Chung WK, McGovern VL, Hevner RF, Conces M, Pierson CR, Scoto M, Muntoni F, Mendell JR, Foust KD. Thomsen G, et al. Nat Med. 2021 Oct;27(10):1701-1711. doi: 10.1038/s41591-021-01483-7. Epub 2021 Oct 4. Nat Med. 2021. PMID: 34608334 Clinical Trial.
Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec (formerly AVXS-101) is a gene therapy that restores SMN production via one-time systemic …
Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletio …
221 results