-
Onasemnogene abeparvovec, sold
under the
brand name Zolgensma, is a gene
therapy used to
treat spinal muscular atrophy (SMA), a
disease causing muscle...
-
disease include nusinersen, risdiplam, and the gene
therapy medication onasemnogene abeparvovec.
Supportive care
includes physical therapy, occupational...
- 2018 for USD 8.7 billion. In 2019, AveXis's
first gene
therapy drug
onasemnogene abeparvovec (Zolgensma)
received regulatory approval in the
United States...
- this
treatment is US$850,000 for both eyes. In May, the FDA
approved onasemnogene abeparvovec (Zolgensma) for
treating spinal muscular atrophy in children...
- 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...
-
Nadofaragene firadenovec (Adstiladrin):
treatment for
bladder cancer Onasemnogene abeparvovec (Zolgensma): AAV-based
treatment for
spinal muscular atrophy...
-
approval granted 2021). In vivo
viral vector-mediated gene therapy:
Onasemnogene Abeparvovec (branded as Zolgesma) for the
treatment of
spinal muscular...
