Patisiran
- Generic Name
- Patisiran
- Brand Names
- Onpattro
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1420706-45-1
- Unique Ingredient Identifier
- 50FKX8CB2Y
- Background
Parisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis . It is marketed as Onpattro which is formulated as patisiran within a liposome envelope for better delivery to the liver, where transthyretin is produced. The approval for Onpattro was granted to Alnyl...
- Indication
适用于治疗成人遗传性转甲状腺素介导的淀粉样变性的多发性神经病，如转甲状腺素蛋白淀粉样变性多发性神经病（ATTR-PN）或转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM）。ATTR疾病也属于罕见病。
- Associated Conditions
- Hereditary transthyretin-mediated amyloidosis
- Associated Therapies
- -
Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
- First Posted Date
- 2022-08-18
- Last Posted Date
- 2024-02-16
- Lead Sponsor
- Alnylam Pharmaceuticals
- Registration Number
- NCT05505838
- Locations
- 🇺🇸
Columbia University Irving Medical Center, New York, New York, United States
🇺🇸Washington University, Saint Louis, Missouri, United States
🇺🇸Mayo Clinic Florida, Jacksonville, Florida, United States
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
- Conditions
- Interventions
- First Posted Date
- 2021-08-27
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Austin Neuromuscular Center
- Target Recruit Count
- 10
- Registration Number
- NCT05023889
- Locations
- 🇺🇸
Austin Neuromuscler Center/National Neuromuscular Research Institute, Austin, Texas, United States
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
- Conditions
- Interventions
- First Posted Date
- 2019-12-17
- Last Posted Date
- 2022-06-06
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 67
- Registration Number
- NCT04201418
- Locations
- 🇺🇸
Clinical Trial Site, Milwaukee, Wisconsin, United States
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
- Conditions
- Interventions
- First Posted Date
- 2019-06-25
- Last Posted Date
- 2024-12-09
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 360
- Registration Number
- NCT03997383
- Locations
- 🇬🇧
Clinical Trial Site, Stockton-on-Tees, United Kingdom
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
- Conditions
- Interventions
- First Posted Date
- 2019-03-05
- Last Posted Date
- 2024-04-22
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 24
- Registration Number
- NCT03862807
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
- Conditions
- Interventions
- First Posted Date
- 2018-11-30
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 164
- Registration Number
- NCT03759379
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
- First Posted Date
- 2015-07-29
- Last Posted Date
- 2023-12-06
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 211
- Registration Number
- NCT02510261
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
- First Posted Date
- 2012-06-13
- Last Posted Date
- 2024-04-19
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 29
- Registration Number
- NCT01617967
- Locations
- 🇸🇪
Clinical Trial Site, Umeå, Sweden
Drug Development Updates
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