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Disease

Familial amyloid neuropathy

Also known as ATTRv amyloidosis, Corino de Andrade's disease, TTR amyloidosis, amyloid neuropathies, familial+12 more

ATTRv amyloidosis, Corino de Andrade's disease, TTR amyloidosis, amyloid neuropathies, familial, amyloidosis, hereditary, transthyretin-related, familial TTR-related amyloidosis, familial amyloid polyneuropathy, familial transthyretin amyloidosis, familial transthyretin-related amyloidosis, hATTR, hereditary TTR amyloid polyneuropathy, hereditary TTR amyloidosis, hereditary transthyretin amyloid polyneuropathy, paramyloidosis, transthyretin-related hereditary amyloidosis, hereditary amyloidosis, transthyretin-related.

1
Associated genes
1
Related proteins

What's happening now

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

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Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Diflunisalapproved

Approval — Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (… (2025)

Approval — Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with… (2025)

Approval — Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult p… (2022)

Accelerated approval — Treatment of stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthy… (2018)

Accelerated approval — Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis… (2018)

Tafamidisapproved

Approval — Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients wi… (2011)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2025emaApprovalDiflunisal· Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2025emaApprovalEplontersen sodium· Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2022emaApprovalVutrisiran sodium· Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2018emaAccelerated approvalPatisiran sodium· Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2018emaAccelerated approvalInotersen sodium· Treatment of stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). source ↗
2011emaApprovalTafamidis· Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage-1 symptomatic polyneuropathy to delay peripheral neurologic impairment. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

1 match

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Reference

Authoritative identity, definition & identifiers.

Synonyms

ATTRv amyloidosis, Corino de Andrade's disease, TTR amyloidosis, amyloid neuropathies, familial, amyloidosis, hereditary, transthyretin-related, familial TTR-related amyloidosis, familial amyloid polyneuropathy, familial transthyretin amyloidosis, familial transthyretin-related amyloidosis, hATTR, hereditary TTR amyloid polyneuropathy, hereditary TTR amyloidosis, hereditary transthyretin amyloid polyneuropathy, paramyloidosis, transthyretin-related hereditary amyloidosis, hereditary amyloidosis, transthyretin-related

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.