Hereditary Transthyretin-mediated Amyloidosis
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
Clinical trial update indicates that Alnylam's patisiran-LNP pregnancy surveillance program is currently recruiting participants for heredit…
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CLINICAL_TRIALS research · 2026-04-02Clinical Trial Update: Phenotypic Manifestations of Hereditary ATTR Amyloidosis
A clinical trial to characterize the phenotypic manifestations of hereditary transthyretin-mediated amyloidosis; it is currently recruiting …
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CLINICAL_TRIALS trial_result · 2026-03-12Clinical Trial Update: Early Detection of Neuropathy in ATTRv
A University of Pennsylvania-sponsored clinical trial on early detection of neuropathy in hereditary transthyretin-mediated amyloidosis (ATT…
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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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CLINICAL_TRIALS research · 2025-08-14Clinical Trial Update: Prospective Evaluation of NfL as a Biomarker in ATTRv
This clinical trial update reports a prospective evaluation of neurofilament light chain (NfL) as a biomarker in hereditary transthyretin-me…
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CLINICAL_TRIALS research · 2025-04-06Clinical Trial Update: Polyneuropathy, Impairments and Physical Activity - The PolyImPAct Study
A clinical trial update for the PolyImPAct study investigating polyneuropathy, impairments, and physical activity, currently recruiting and …
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EMA approval · 2025-03-25EMA Authorised: Onpattro (patisiran sodium) — Alnylam Netherlands B.V.
The European Medicines Agency authorised Onpattro (patisiran) for familial hereditary transthyretin-mediated amyloidosis with orphan designa…
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FDA approval · 2025-03-20FDA Approved: AMVUTTRA — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
FDA approved a supplemental application for AMVUTTRA (vutrisiran) submitted by Alnylam Pharmaceuticals, expanding the approved labeling for …
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CLINICAL_TRIALS research · 2024-05-20Clinical Trial Update: Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Expanded access protocol for patisiran in hereditary transthyretin-mediated amyloidosis has been approved for marketing, enabling broader pa…
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CLINICAL_TRIALS trial_result · 2022-12-19Clinical Trial Update: Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ). [Phase 1, Phase 2]
Phase 1/2 trial evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple subcutaneous doses of eplonte…
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FDA approval · 2021-05-06FDA Approved: ONPATTRO — Supplemental Application (Alnylam Pharmaceuticals, Inc.)
FDA approved a supplemental application for ONPATTRO (patisiran) by Alnylam Pharmaceuticals.
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CLINICAL_TRIALS trial_result · 2015-08-24Clinical Trial Update: The Effect of Diflunisal on Familial Transthyretin Amyloidosis
Completed clinical trial evaluating the NSAID diflunisal for hereditary transthyretin-mediated amyloidosis (familial transthyretin amyloidos…