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7. 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]
8. 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]
9. [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]
10. 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]
11. Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology. Hordeaux J; Buza EL; Dyer C; Goode T; Mitchell TW; Richman L; Denton N; Hinderer C; Katz N; Schmid R; Miller R; Choudhury GR; Horiuchi M; Nambiar K; Yan H; Li M; Wilson JM Hum Gene Ther; 2020 Aug; 31(15-16):808-818. PubMed ID: 32845779 [TBL] [Abstract][Full Text] [Related]
12. 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]
13. 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]
14. 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]
15. 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]
16. Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec. Retson L; Tiwari N; Vaughn J; Bernes S; Adelson PD; Mansfield K; Libertini S; Kuzmiski B; Alecu I; Gabriel R; Mangum R Mol Ther; 2023 Oct; 31(10):2991-2998. PubMed ID: 37598295 [TBL] [Abstract][Full Text] [Related]
17. Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec. Day JW; Finkel RS; Mercuri E; Swoboda KJ; Menier M; van Olden R; Tauscher-Wisniewski S; Mendell JR Mol Ther Methods Clin Dev; 2021 Jun; 21():76-82. PubMed ID: 33768131 [TBL] [Abstract][Full Text] [Related]
18. Onasemnogene Abeparvovec: First Global Approval. Hoy SM Drugs; 2019 Jul; 79(11):1255-1262. PubMed ID: 31270752 [TBL] [Abstract][Full Text] [Related]
19. Onasemnogene abeparvovec gene therapy for spinal muscular atrophy: A cohort study from the United Arab Emirates. Mundada V; Narayan O; Arora S; Beri N; Abusamra R; Mullasery D; Parashar D Muscle Nerve; 2024 Oct; 70(4):808-815. PubMed ID: 39087519 [TBL] [Abstract][Full Text] [Related]
20. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy. Chand D; Mohr F; McMillan H; Tukov FF; Montgomery K; Kleyn A; Sun R; Tauscher-Wisniewski S; Kaufmann P; Kullak-Ublick G J Hepatol; 2021 Mar; 74(3):560-566. PubMed ID: 33186633 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]