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4. 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]
5. 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]
6. Novel use of nusinersen as a therapeutic bridge to onasemnogene abeparvovec-xioi in a premature neonate with type 1 spinal muscular atrophy. Ferrante L; Melendez-Zaidi A; Lindsey W; Lotze T Muscle Nerve; 2022 Aug; 66(2):E8-E10. PubMed ID: 35616550 [No Abstract] [Full Text] [Related]
7. 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]
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9. How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison. Ribero VA; Daigl M; Martí Y; Gorni K; Evans R; Scott DA; Mahajan A; Abrams KR; Hawkins N J Comp Eff Res; 2022 Apr; 11(5):347-370. PubMed ID: 35040693 [No Abstract] [Full Text] [Related]
10. 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]
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16. Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial. Shell RD; McGrattan KE; Hurst-Davis R; Young SD; Baranello G; Lavrov A; O'Brien E; Wallach S; LaMarca N; Reyna SP; Darras BT Neuromuscul Disord; 2023 Aug; 33(8):670-676. PubMed ID: 37455203 [TBL] [Abstract][Full Text] [Related]
17. Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio. Waldrop MA; Chagat S; Storey M; Meyer A; Iammarino M; Reash N; Alfano L; Lowes L; Noritz G; Prochoroff A; Rossman I; Ginsberg M; Mosher K; Broomall E; Bass N; Gushue C; Kotha K; Paul G; Shell R; Tsao CY; Mendell JR; Connolly AM Neuromuscul Disord; 2024 Jan; 34():41-48. PubMed ID: 38142474 [TBL] [Abstract][Full Text] [Related]
18. 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]
19. 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]
20. Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study. Stettner GM; Hasselmann O; Tscherter A; Galiart E; Jacquier D; Klein A BMC Neurol; 2023 Feb; 23(1):88. PubMed ID: 36855136 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]