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Alnylam Pharmaceuticals — PHASE3
AMVUTTRA: FDA approved
treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits
Attruby: FDA approved
treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization
WAINUA: FDA approved
Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Onpattro: FDA approved
ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
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The Assistance Fund — Hereditary transthyretin-mediated amyloidosis
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AMVUTTRA
(VUTRISIRAN)Orphan drugAlnylam Pharmaceuticals, Inc.
Attruby
(ACORAMIDIS HYDROCHLORIDE)Orphan drugBridgeBio Pharma, Inc.
WAINUA
(EPLONTERSEN)Orphan drugAstraZeneca Pharmaceuticals LP
Onpattro
(PATISIRAN)Orphan drugAlnylam Pharmaceuticals, Inc.
Vyndaqel
(TAFAMIDIS MEGLUMINE)Orphan drugstandardPfizer Laboratories Div Pfizer Inc
Therapeutic Efficacy and Safety Profile of Eplontersen in Hereditary Transthyretin-Mediated Amyloidosis: A Systematic Review.
Hereditary transthyretin-mediated amyloidosis (hATTR) is a disorder that affects several body systems and can result in life-threatening conditions like cardiomyopathy and polyneuropathy. For treatmen...
Efficacy and Safety of Vutrisiran in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: Analysis of the East Asian Subpopulation from HELIOS-A.
In the phase 3 HELIOS-A study (NCT03759379), vutrisiran significantly improved a range of disease-related endpoints compared with an external placebo in patients with hereditary transthyretin amyloido...
Neuropathy impairment and nutritional status with eplontersen in patients with hereditary transthyretin-mediated amyloidosis.
Neuropathy impairment and nutritional status with eplontersen in patients with hereditary transthyretin-mediated amyloidosis.
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Laura Obici
Amyloidosis Research and Treatment Centre
Michael Polydefkis, M.D
Johns Hopkins Hospital
📍 BALTIMORE, MD
Chongshu Chen
Alnylam Pharmaceuticals
Chi-Chao Chao
National Taiwan University Hospital
Violaine Planté-Bordeneuve
East Paris University
David Adams
Centre Hospitalier Universitaire (CHU) Bicêtre
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