Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

首次发布于 2022 年 8 月 18 日

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

计划入组 2,000 人 · 首次发布于 2026 年 1 月 22 日

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

计划入组 1,750 人 · 首次发布于 2025 年 7 月 8 日

Transthyretin Amyloidosis Therapy (HuidaGene)

TTR gene therapy (transthyretin amyloidosis)

CRISPR-Cas12 gene therapy (HG-PRECISE, Transthyretin Amyloidosis)

Lexeo Therapeutics

United States

MiNK Therapeutics

United States

Mesoblast

United States

BridgeBio to Present Cardiovascular Mortality Data for Acoramidis at ESC Congress 2025

FDA Approves Alnylam's AMVUTTRA for ATTR Cardiomyopathy, Sending Stock Soaring

EU Approves WAINZUA (Eplontersen) for Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy