Amyloidosis, Familial
Recent clinical, regulatory, research and industry developments relating to this disease.
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 1 clinical trial expected to report results, the earliest in Q3 2027.
Clinical MilestonesViewHide
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
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
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
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.
Diseases in which there is a familial pattern of AMYLOIDOSIS.
- 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.