Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle...
33 KB (2,443 words) - 02:11, 16 July 2024
tisagenlecleucel (2017), voretigene neparvovec (2017), patisiran (2018), onasemnogene abeparvovec (2019), idecabtagene vicleucel (2021), nadofaragene firadenovec...
176 KB (18,162 words) - 17:07, 18 October 2024
include nusinersen, risdiplam, and the gene therapy medication onasemnogene abeparvovec. Supportive care includes physical therapy, occupational therapy...
89 KB (9,948 words) - 05:18, 19 October 2024
Novartis Gene Therapies. In 2019, AveXis's first gene therapy drug onasemnogene abeparvovec (Zolgensma) received regulatory approval in the United States and...
10 KB (784 words) - 22:30, 12 September 2024
M09AX06 Eteplirsen M09AX07 Nusinersen M09AX08 Golodirsen M09AX09 Onasemnogene abeparvovec M09AX10 Risdiplam M09AX11 Palovarotene M09AX12 Viltolarsen M09AX13...
1 KB (248 words) - 05:17, 18 December 2023
Intravenous, intrathecal I–III 150+ Several ongoing and complete (onasemnogene abeparvovec) Congestive heart failure SERCA2a Intra-coronary IIb 250 Ongoing...
72 KB (8,401 words) - 06:44, 28 August 2024
were used in several clinically approved gene therapies including onasemnogene abeparvovec which uses AAV9. Wilson is the founder of several gene therapy...
11 KB (898 words) - 17:24, 1 August 2024
Nadofaragene firadenovec (Adstiladrin): treatment for bladder cancer Onasemnogene abeparvovec (Zolgensma): AAV-based treatment for spinal muscular atrophy Strimvelis:...
10 KB (792 words) - 10:27, 12 December 2023
approval granted 2021). In vivo viral vector-mediated gene therapy: Onasemnogene Abeparvovec (branded as Zolgesma) for the treatment of spinal muscular atrophy...
78 KB (8,788 words) - 19:06, 6 August 2024