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5. Zolgensma - one-time gene therapy for spinal muscular atrophy. Med Lett Drugs Ther; 2019 Jul; 61(1577):113-114. PubMed ID: 31381549 [No 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. 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]
8. [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]
9. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy. Kirschner J; Butoianu N; Goemans N; Haberlova J; Kostera-Pruszczyk A; Mercuri E; van der Pol WL; Quijano-Roy S; Sejersen T; Tizzano EF; Ziegler A; Servais L; Muntoni F Eur J Paediatr Neurol; 2020 Sep; 28():38-43. PubMed ID: 32763124 [TBL] [Abstract][Full Text] [Related]
10. Onasemnogene Abeparvovec: First Global Approval. Hoy SM Drugs; 2019 Jul; 79(11):1255-1262. PubMed ID: 31270752 [TBL] [Abstract][Full Text] [Related]
11. Nusinersen in the Treatment of Spinal Muscular Atrophy. Goodkey K; Aslesh T; Maruyama R; Yokota T Methods Mol Biol; 2018; 1828():69-76. PubMed ID: 30171535 [TBL] [Abstract][Full Text] [Related]
13. 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]
14. [Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM)]. Ziegler A; Wilichowski E; Schara U; Hahn A; Müller-Felber W; Johannsen J; von der Hagen M; von Moers A; Stoltenburg C; Saffari A; Walter MC; Husain RA; Pechmann A; Köhler C; Horber V; Schwartz O; Kirschner J Nervenarzt; 2020 Jun; 91(6):518-529. PubMed ID: 32394004 [TBL] [Abstract][Full Text] [Related]
15. 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]
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17. 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]
18. Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series. Gaillard J; Gu AR; Neil Knierbein EE J Pediatr; 2023 Sep; 260():113493. PubMed ID: 37211209 [TBL] [Abstract][Full Text] [Related]