Hereditary neuropathic amyloidosis

2 trials 1 recruiting MedDRA PT
2 Registered trials
1 Recruiting
2 Unique sponsors
3 Unique IMPs
2 Commercial trials
Drug classes (ATC)
— ATC code not specified 1
N Neurology/Psychiatry (ATC N) 2
Mechanism of action (INN-stem analysis)
siRNA 2 substances
Unknown / small molecule 1 substance
Phase III: 2 Phase II/III: 1

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Show: 2 trials

Phase III

2 trials
  • Alnylam Pharmaceuticals Inc. Commercial
    PATISIRAN; VUTRISIRAN
    2023-508365-33-00 7 countries 2019 n=44 Orphan
    Ended
  • Alnylam Pharmaceuticals Inc. Academic
    PATISIRAN; VUTRISIRAN
    2023-508365-33-00 7 countries 2019 n=44 Orphan
    Ended

Phase II/III

1 trial
  • Intellia Therapeutics Inc. Commercial
    ZICLUMERAN, SINGLE GUIDE RNA TARGETING THE HUMAN TTR GENE
    2023-507385-11-00 2 countries 2024 n=15 Orphan
    Recruiting
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Alnylam Pharmaceuticals Inc. Commercial 2 Phase III 0
Intellia Therapeutics Inc. Commercial 1 Phase II/III 1
New trials by year
2019
2
2024
1
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