Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy [Phase 3]
A Phase 3 trial (TRITON-PN) evaluating Nucresiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy is currently recruit…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: A Phase 3 Study of NTLA-2001 in ATTRv-PN [Phase 3]
NTLA-2001, Intellia's in vivo gene-editing therapy, is in Phase 3 for hereditary transthyretin-mediated amyloidosis with polyneuropathy and …
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CLINICAL_TRIALS research · 2025-12-24Clinical Trial Update: Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
An observational study is evaluating neurofilament light chain as a biomarker in asymptomatic carriers of transthyretin variants and in pati…
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EMA approval · 2025-11-05EMA Authorised: Beyonttra (acoramidis hydrochloride) — Bayer AG
The European Medicines Agency authorised Beyonttra (acoramidis hydrochloride) by Bayer AG for hereditary transthyretin-mediated amyloidosis …
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CLINICAL_TRIALS trial_result · 2025-08-19Clinical Trial Update: TEGSEDI Pregnancy Surveillance Program
Clinical trial update shows a TEGSEDI pregnancy surveillance program for hereditary transthyretin-mediated amyloidosis with polyneuropathy i…
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EMA approval · 2025-08-13EMA Authorised: Amvuttra (vutrisiran sodium) — Alnylam Netherlands B.V.
EMA authorised Amvuttra (vutrisiran) for hereditary transthyretin-mediated amyloidosis with polyneuropathy (familial amyloid neuropathy).
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CLINICAL_TRIALS research · 2025-06-29Clinical Trial Update: TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
This clinical trial update describes the TRAMmoniTTR study, a genetic screening program for individuals at risk of hereditary transthyretin …
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FDA approval · 2025-03-11FDA Approved: AMVUTTRA — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved a supplemental application for Amvuttra (vutrisiran) submitted by Alnylam Pharmaceuticals, reflecting a regulatory update t…
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FDA approval · 2023-01-13FDA Approved: ONPATTRO — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved a supplemental application for ONPATTRO (patisiran) from Alnylam Pharmaceuticals, reflecting a regulatory action to modify …
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FDA approval · 2022-10-03FDA Approved: ONPATTRO — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
FDA approved a supplemental application for ONPATTRO (patisiran) by Alnylam Pharmaceuticals.
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FDA approval · 2022-06-13FDA Approved: AMVUTTRA — Original Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved AMVUTTRA (vutrisiran) from Alnylam Pharmaceuticals for hereditary transthyretin-mediated amyloidosis with polyneuropathy. T…
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FDA approval · 2020-04-17FDA Approved: ONPATTRO — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved a supplemental application for ONPATTRO (patisiran) from Alnylam Pharmaceuticals.
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FDA approval · 2019-09-16FDA Approved: ONPATTRO — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved a supplemental application for ONPATTRO (patisiran) submitted by Alnylam Pharmaceuticals, Inc., indicating an update to its…
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FDA approval · 2019-05-06FDA Approved: ONPATTRO — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved a supplemental application for ONPATTRO (patisiran) submitted by Alnylam Pharmaceuticals.
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FDA approval · 2018-08-10FDA Approved: ONPATTRO — Original Application (Alnylam Pharmaceuticals, Inc.)
The FDA approved ONPATTRO (patisiran) as the original application for hereditary transthyretin-mediated amyloidosis with polyneuropathy, dev…