-
Onasemnogene abeparvovec, sold
under the
brand name Zolgensma, is a gene
therapy used to
treat spinal muscular atrophy (SMA), a
disease causing muscle...
- nusinersen, risdiplam, and the gene
therapy medication onasemnogene abeparvovec.
Supportive care
includes physical therapy,
occupational therapy, respiratory...
- Gene Therapies. In 2019, AveXis's
first gene
therapy drug
onasemnogene abeparvovec (Zolgensma)
received regulatory approval in the
United States and, with...
- (2017),
voretigene neparvovec (2017),
patisiran (2018),
onasemnogene abeparvovec (2019),
idecabtagene vicleucel (2021),
nadofaragene firadenovec, valoctocogene...
- Intravenous,
intrathecal I–III 150+
Several ongoing and
complete (onasemnogene
abeparvovec)
Congestive heart failure SERCA2a Intra-coronary IIb 250 Ongoing...
-
Eteplirsen M09AX07
Nusinersen M09AX08
Golodirsen M09AX09
Onasemnogene abeparvovec M09AX10
Risdiplam M09AX11
Palovarotene M09AX12 Viltol****n M09AX13 Casimersen...
- (USAN)
onaclostox onamelatucel-L (USAN)
onapristone (INN)
onasemnogene abeparvovec (USAN, INN)
Oncaspar Oncovin ondansetron (INN)
onercept (USAN) Onoact...
-
autoleucel (Aucatzyl):
treatment of
acute lymphoblastic leukemia Onasemnogene abeparvovec (Zolgensma): AAV-based
treatment for
spinal muscular atrophy Strimvelis:...
- in
several clinically approved gene
therapies including onasemnogene abeparvovec which uses AAV9.
Wilson is the
founder of
several gene
therapy companies...
-
granted 2021). In vivo
viral vector-mediated gene therapy:
Onasemnogene Abeparvovec (branded as Zolgesma) for the
treatment of
spinal muscular atrophy, administered...
