Disease

Amyloidosis, Familial

Late-stage therapeutic developmentEmerging research

MeSHAmyloidosis, Familial

Publications

Clinical trials

2023

Latest publication

Latest activity

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Recent clinical, regulatory, research and industry developments relating to this disease.

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.

Research2023-10-01The New England journal of medicine

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low

Key developments

1 clinical trial expected to report results, the earliest in Q3 2027.

Upcoming Milestones

Q3 2027MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Major developments

Upcoming trial readoutHigh impact

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Results expected Q3 20272026-04-16

Clinical Milestones1View

Results expected 1

2026-04-16MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)Results expected Q3 2027

Activity timeline1

2026-04-16ClinicalMAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)Results expected Q3 2027

Clinical trials

2 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

Phase 3 · Recruiting · Intellia Therapeutics

An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant

Phase 3 · Completed · Alnylam Pharmaceuticals

Recruiting

Phase 3 · Recruiting · Intellia Therapeutics

Recently completed

Phase 3 · Completed · Alnylam Pharmaceuticals

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Most influential

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.

The New England journal of medicine · 2023 · 230 cites

Recent publications

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.

The New England journal of medicine · 2023 · 230 cites

Major themes4

Amyloidosis1
Cardiomyopathies1
Prealbumin1
RNA, Small Interfering1

Leading journals1

The New England journal of medicine1

Leading researchers8

Badri P1
Berk JL1
Chen J1
Damy T1
Di Carli M1
Diemberger I1
Donal E1
Fernandes F1

Affiliations (unnormalised)1

From Columbia University Irving Medical Center (M.S.M.) and Grossman School of Medicine1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Diseases in which there is a familial pattern of AMYLOIDOSIS.

Identifiers

MeSHAmyloidosis, Familial

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
Related entities are derived from literature co-mention (studied together) — associative, not causal.