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5. 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]
6. 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]
7. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. Mendell JR; Al-Zaidy SA; Lehman KJ; McColly M; Lowes LP; Alfano LN; Reash NF; Iammarino MA; Church KR; Kleyn A; Meriggioli MN; Shell R JAMA Neurol; 2021 Jul; 78(7):834-841. PubMed ID: 33999158 [TBL] [Abstract][Full Text] [Related]
8. Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes. Waldrop MA; Karingada C; Storey MA; Powers B; Iammarino MA; Miller NF; Alfano LN; Noritz G; Rossman I; Ginsberg M; Mosher KA; Broomall E; Goldstein J; Bass N; Lowes LP; Tsao CY; Mendell JR; Connolly AM Pediatrics; 2020 Sep; 146(3):. PubMed ID: 32843442 [TBL] [Abstract][Full Text] [Related]
9. 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]
11. 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]
12. 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]
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. [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]
15. Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy. Blair HA CNS Drugs; 2022 Sep; 36(9):995-1005. PubMed ID: 35960489 [TBL] [Abstract][Full Text] [Related]
16. 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]
17. 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]