-
Patisiran, sold
under the
brand name Onpattro, is a
medication used for the
treatment of
polyneuropathy in
people with
hereditary transthyretin-mediated...
- In late 2016, the company's lead
candidate in
phase III
studies was
patisiran, a
treatment targeting transthyretin (TTR) for the
treatment of TTR-mediated...
-
European Union and in the USA.
Patisiran functions similarly to inotersen. Moderate-certainty
evidence suggests that
patisiran mitigates worsening of peripheral...
-
transthyretin familial amyloid polyneuropathy. In
August 2018, the FDA
approved patisiran, an siRNA-based treatment, at an
expected cost of up to $450,000 per year...
-
Strimvelis (2016),
tisagenlecleucel (2017),
voretigene neparvovec (2017),
patisiran (2018),
onasemnogene abeparvovec (2019),
idecabtagene vicleucel (2021)...
- disorders.
Patisiran is the
first double stranded siRNA-based
medication approved in 2018 and
developed by
Alnylam Pharmaceuticals.
Patisiran uses the RNAi...
-
vutrisiran to
patisiran. 164
ATTRv amyloidosis patients were
randomized 3:1 to
subcutaneous vutrisiran 25 mg
every 3
months or
patisiran 0.3 mg/kg IV infusion...
-
phospholipase 2G6-****ociated
neurodegeneration (PLAN). The FDA
granted Patisiran (Onpattro)
orphan drug
status and
breakthrough therapy designation due...
- and
medications that
decreases the
activity of the
transthyretin genes (
patisiran and inotersen). In
patients with
familial transthyretin mutations, liver...
-
first company to have a
siRNA therapy approved by the FDA.
Onpattro (
patisiran) was
approved for the
treatment of
polyneuropathy of
hereditary transthyretin-mediated...
