Dystonia Coalition Projects

计划入组 2 人 · 首次发布于 2019 年 11 月 7 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

Congress's Failure to Renew Priority Review Voucher Program Threatens Rare Disease Drug Development

US House Passes Bipartisan Bill to Reauthorize FDA's Rare Pediatric Disease Priority Review Voucher Program

Exempting Rare Disease Drugs from Medicare Pricing Pilots Would Erase Most Retail Savings, Harvard Analysis Finds