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6. Onasemnogene abeparvovec for the treatment of spinal muscular atrophy. McMillan HJ; Proud CM; Farrar MA; Alexander IE; Muntoni F; Servais L Expert Opin Biol Ther; 2022 Sep; 22(9):1075-1090. PubMed ID: 35437095 [TBL] [Abstract][Full Text] [Related]
7. [Pharmacological and clinical profile of Onasemnogene Aveparvovec, the first gene therapy for spinal muscular atrophy (SMA)]. Atsumi A; Yoneda T; Tsuchida K; Kagawa Y; Tominaga S; Kawase K; Kikuchi N Nihon Yakurigaku Zasshi; 2022; 157(1):53-61. PubMed ID: 34980814 [TBL] [Abstract][Full Text] [Related]
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11. Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program. Chand DH; Mitchell S; Sun R; LaMarca N; Reyna SP; Sutter T Pediatr Neurol; 2022 Jul; 132():27-32. PubMed ID: 35605311 [TBL] [Abstract][Full Text] [Related]
12. Onasemnogene Abeparvovec: First Global Approval. Hoy SM Drugs; 2019 Jul; 79(11):1255-1262. PubMed ID: 31270752 [TBL] [Abstract][Full Text] [Related]
13. 2024 update: European consensus statement on gene therapy for spinal muscular atrophy. Kirschner J; Bernert G; Butoianu N; De Waele L; Fattal-Valevski A; Haberlova J; Moreno T; Klein A; Kostera-Pruszczyk A; Mercuri E; Quijano-Roy S; Sejersen T; Tizzano EF; van der Pol WL; Wallace S; Zafeiriou D; Ziegler A; Muntoni F; Servais L Eur J Paediatr Neurol; 2024 Jul; 51():73-78. PubMed ID: 38878702 [TBL] [Abstract][Full Text] [Related]
14. 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 Cureus; 2023 Mar; 15(3):e36197. PubMed ID: 37065340 [TBL] [Abstract][Full Text] [Related]
15. Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 1. Bischof M; Lorenzi M; Lee J; Druyts E; Balijepalli C; Dabbous O Curr Med Res Opin; 2021 Oct; 37(10):1719-1730. PubMed ID: 34236007 [TBL] [Abstract][Full Text] [Related]
17. Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry. Servais L; Day JW; De Vivo DC; Kirschner J; Mercuri E; Muntoni F; Proud CM; Shieh PB; Tizzano EF; Quijano-Roy S; Desguerre I; Saito K; Faulkner E; Benguerba KM; Raju D; LaMarca N; Sun R; Anderson FA; Finkel RS J Neuromuscul Dis; 2024; 11(2):425-442. PubMed ID: 38250783 [TBL] [Abstract][Full Text] [Related]
18. Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec -A Single Centre Experience. Friese J; Geitmann S; Holzwarth D; Müller N; Sassen R; Baur U; Adler K; Kirschner J J Neuromuscul Dis; 2021; 8(2):209-216. PubMed ID: 33427694 [TBL] [Abstract][Full Text] [Related]
19. Onasemnogene abeparvovec-xioi: a gene replacement strategy for the treatment of infants diagnosed with spinal muscular atrophy. Schwartz M; Likhite S; Meyer K Drugs Today (Barc); 2021 Jun; 57(6):387-399. PubMed ID: 34151905 [TBL] [Abstract][Full Text] [Related]
20. Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: A systematic review and meta-analysis. Yang D; Ruan Y; Chen Y J Paediatr Child Health; 2023 Mar; 59(3):431-438. PubMed ID: 36722610 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]