National Registry of Rare Kidney Diseases

计划入组 35,000 人 · 首次发布于 2023 年 10 月 4 日

Swiss Rare Disease Registry (SRDR)

计划入组 500,000 人 · 首次发布于 2022 年 1 月 5 日

AI-assisted Rare Disease Diagnosis

计划入组 1,056 人 · 首次发布于 2026 年 6 月 16 日

undisclosed rare disease therapeutic (rare disease/eye disease/skin disease)

undisclosed pediatric rare disease therapeutic (rare disease/kidney disease)

undisclosed pediatric rare disease therapeutic (rare disease/immune disease)

Rare Disease Research, LLC

CURE RARE DISEASE, INC.

United States

Rare Disease Therapeutics, Inc.

United States

Monopar Acquires Late-Stage Wilson Disease Drug Candidate ALXN-1840 from Alexion, AstraZeneca Rare Disease

Monopar Acquires Late-Stage Wilson Disease Drug Candidate ALXN-1840 from Alexion

Alexion Data Show Ultomiris and Soliris Reduce Steroid Use and Improve Outcomes in gMG