-
Onasemnogene abeparvovec, sold
under the
brand name Zolgensma, is a gene
therapy used to
treat spinal muscular atrophy (SMA), a
disease causing muscle...
- to
Novartis Gene Therapies. In 2019, AveXis's
first gene
therapy drug
onasemnogene abeparvovec (Zolgensma)
received regulatory approval in the
United States...
-
disease include nusinersen, risdiplam, and the gene
therapy medication onasemnogene abeparvovec.
Supportive care
includes physical therapy, occupational...
-
tisagenlecleucel (2017),
voretigene neparvovec (2017),
patisiran (2018),
onasemnogene abeparvovec (2019),
idecabtagene vicleucel (2021),
nadofaragene firadenovec...
- acid M09AX06
Eteplirsen M09AX07
Nusinersen M09AX08
Golodirsen M09AX09
Onasemnogene abeparvovec M09AX10
Risdiplam M09AX11
Palovarotene M09AX12 Viltol****n...
- SMN1 Intravenous,
intrathecal I–III 150+
Several ongoing and
complete (
onasemnogene abeparvovec)
Congestive heart failure SERCA2a Intra-coronary IIb 250...
-
onabotulinumtoxinA (USAN)
onaclostox onamelatucel-L (USAN)
onapristone (INN)
onasemnogene abeparvovec (USAN, INN)
Oncaspar Oncovin ondansetron (INN)
onercept (USAN)...
-
Obecabtagene autoleucel (Aucatzyl):
treatment of
acute lymphoblastic leukemia Onasemnogene abeparvovec (Zolgensma): AAV-based
treatment for
spinal muscular atrophy...
- lab were used in
several clinically approved gene
therapies including onasemnogene abeparvovec which uses AAV9.
Wilson is the
founder of
several gene therapy...
-
approval granted 2021). In vivo
viral vector-mediated gene therapy:
Onasemnogene Abeparvovec (branded as Zolgesma) for the
treatment of
spinal muscular...
