Hereditary ATTR amyloidosis Advocacy Hub | UniteRare

At a Glance

Live from database

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Regulatory Watchboard

5 events

Apr 2026WAINUA: New indication approved

FDAcompleted

Dec 2025WAINUA: New indication approved

FDAcompleted

Apr 2025WAINUA: New indication approved

FDAcompleted

Sep 2024WAINUA: New indication approved

FDAcompleted

Dec 2023WAINUA: FDA approved

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

3 programs

FINANCIAL LANDSCAPE SUMMARY

Total programs

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Copay cards

Copay Assistance2

Vyndaqel

Pfizer Laboratories Div Pfizer Inc

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Tegsedi

Ionis/AstraZeneca

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Patient Assistance Programs1

WAINUA

AstraZeneca Pharmaceuticals LP

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Approved Treatments

5 FDA-approved

Tegsedi

(inotersen)Orphan drug

Ionis/AstraZeneca

Approved Oct 2018

Vyndaqel

(TAFAMIDIS MEGLUMINE)Orphan drug

Pfizer Laboratories Div Pfizer Inc

FDA label ↗

WAINUA

(EPLONTERSEN)Orphan drug

AstraZeneca Pharmaceuticals LP

FDA label ↗

Onpattro

(PATISIRAN)Orphan drug

Alnylam Pharmaceuticals, Inc.

FDA label ↗

AMVUTTRA

(VUTRISIRAN)Orphan drug

Alnylam Pharmaceuticals, Inc.

FDA label ↗

View full treatment details on disease page →

Clinical Trial Landscape

No active clinical trials currently recruiting for Hereditary ATTR amyloidosis.
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Recent News & Research

1 article

AlnylamFeb 5, 2026

Alnylam Reports Record $2.8B Revenue from RNAi Portfolio

Alnylam reported $2.8 billion from RNA interference therapies for hereditary ATTR and other rare diseases.

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Browse all Hereditary ATTR amyloidosis news →

Specialist Network

Top 6 by expertise

John L. Berk, MD, D.D.S

Boston University

📍 CASTRO VALLEY, CA

Score 101 trial

John Vest, MD, BAS

Alnylam Pharmaceuticals

📍 COLORADO SPRINGS, CO

Score 71 trial

EMILIE GARRIDO PRADALIE

APHM DRIC

Score 713 trials

Mathew S Maurer, MD

Columbia University

Score 51 trial

Omar F Abou Ezzeddine, MD

Mayo Clinic

Score 31 trial

Erwan Donal

Rennes University Hospital

Score 31 trial21 pubs

View all Hereditary ATTR amyloidosis specialists →

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Financial Assistance

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