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5. Health Care Resource Utilization and Costs for Patients with Spinal Muscular Atrophy: Findings from a Retrospective US Claims Database Analysis. Toro W; Yang M; Georgieva M; Song W; Patel A; Jiang AX; Zhao A; LaMarca N; Dabbous O Adv Ther; 2023 Oct; 40(10):4589-4605. PubMed ID: 37587305 [TBL] [Abstract][Full Text] [Related]
6. Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study. Bitetti I; Lanzara V; Margiotta G; Varone A Gene Ther; 2023 Aug; 30(7-8):592-597. PubMed ID: 35606491 [TBL] [Abstract][Full Text] [Related]
7. 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 Hum Gene Ther; 2023 Feb; 34(3-4):129-138. PubMed ID: 36136906 [TBL] [Abstract][Full Text] [Related]
8. 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 C; Ziegler A; Becker LL; Johannsen J; Brennenstuhl H; Schreiber G; Flotats-Bastardas M; Stoltenburg C; Hartmann H; Illsinger S; Denecke J; Pechmann A; Müller-Felber W; Vill K; Blaschek A; Smitka M; van der Stam L; Weiss K; Winter B; Goldhahn K; Plecko B; Horber V; Bernert G; Husain RA; Rauscher C; Trollmann R; Garbade SF; Hahn A; von der Hagen M; Kaindl AM Lancet Child Adolesc Health; 2022 Jan; 6(1):17-27. PubMed ID: 34756190 [TBL] [Abstract][Full Text] [Related]
9. Gene transfer therapy in children with spinal muscular atrophy: A single-center experience with a cohort of 25 children. Chencheri N; Alexander G; Nugud A; Majadas E; Salim H; Prudhomme K; DeJager N; Janardhanan VS; Elbashir H Muscle Nerve; 2023 Sep; 68(3):269-277. PubMed ID: 37392188 [TBL] [Abstract][Full Text] [Related]
10. [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]
11. 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]
13. 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]
14. 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]
15. Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy. Kichula EA; Proud CM; Farrar MA; Kwon JM; Saito K; Desguerre I; McMillan HJ Muscle Nerve; 2021 Oct; 64(4):413-427. PubMed ID: 34196026 [TBL] [Abstract][Full Text] [Related]
16. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Al-Zaidy SA; Mendell JR Pediatr Neurol; 2019 Nov; 100():3-11. PubMed ID: 31371124 [TBL] [Abstract][Full Text] [Related]
17. Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom. Gowda V; Atherton M; Murugan A; Servais L; Sheehan J; Standing E; Manzur A; Scoto M; Baranello G; Munot P; McCullagh G; Willis T; Tirupathi S; Horrocks I; Dhawan A; Eyre M; Vanegas M; Fernandez-Garcia MA; Wolfe A; Pinches L; Illingworth M; Main M; Abbott L; Smith H; Milton E; D'Urso S; Vijayakumar K; Marco SS; Warner S; Reading E; Douglas I; Muntoni F; Ong M; Majumdar A; Hughes I; Jungbluth H; Wraige E Lancet Reg Health Eur; 2024 Feb; 37():100817. PubMed ID: 38169987 [TBL] [Abstract][Full Text] [Related]
18. Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy. René CA; Parks RJ Pharmaceutics; 2023 Jun; 15(6):. PubMed ID: 37376212 [TBL] [Abstract][Full Text] [Related]
19. 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]