Familial Amyloid Polyneuropathy
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CLINICAL_TRIALS trial_result · 2026-03-12Clinical Trial Update: Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
Completed clinical trial update describing approaches to monitoring early disease progression in hereditary transthyretin amyloidosis. The s…
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EMA approval · 2026-01-27EMA Authorised: Wainzua (eplontersen sodium) — AstraZeneca AB
The European Medicines Agency authorised Wainzua (eplontersen sodium) from AstraZeneca AB for familial amyloid polyneuropathy.
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EMA approval · 2025-12-19EMA Authorised: Attrogy (diflunisal) — Purpose Pharma International AB
The EMA has authorised Attrogy (diflunisal) by Purpose Pharma International AB for familial amyloid polyneuropathy, marking EU marketing aut…
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CLINICAL_TRIALS trial_result · 2023-02-09Clinical Trial Update: Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) [Phase 3]
An extensionPhase 3 trial evaluated long-term safety and efficacy of IONIS-TTR Rx in familial amyloid polyneuropathy, with completed status …
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FDA approval · 2019-05-03FDA Approved: VYNDAQEL — Original Application (Pfizer Laboratories Div Pfizer Inc)
FDA approved VYNDAQEL (tafamidis meglumine) as the original application for the treatment of transthyretin-related familial amyloid polyneur…
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CLINICAL_TRIALS trial_result · 2019-01-23Clinical Trial Update: Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy [Phase 2, Phase 3]
Phase 2/3 trial results summarize efficacy and safety of inotersen for familial amyloid polyneuropathy (FAP).
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CLINICAL_TRIALS trial_result · 2018-09-06Clinical Trial Update: APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis [Phase 3]
Phase 3 study of patisiran (ALN-TTR02) for transthyretin-mediated amyloidosis has completed with results posted in 2018.
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CLINICAL_TRIALS trial_result · 2017-03-17Clinical Trial Update: The Effect of Diflunisal on Familial Amyloidosis [Phase 2, Phase 3]
This update reports the completion and results posting of phase 2 and phase 3 trials evaluating diflunisal for familial amyloid polyneuropat…
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CLINICAL_TRIALS research · 2016-11-30Clinical Trial Update: Study of SOM0226 in Familial Amyloid Polyneuropathy [Phase 1, Phase 2]
A Phase 1/2 clinical trial of SOM0226 in familial amyloid polyneuropathy has completed.
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CLINICAL_TRIALS trial_result · 2012-12-17Clinical Trial Update: Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis [Phase 2, Phase 3]
Pfizer's Fx-1006A completed a Phase 2/3 study in Familial Amyloid Polyneuropathy, with safety and efficacy results posted in 2012.