Rare Genetic Disorders of the Breathing Airways

计划入组 367 人 · 首次发布于 2006 年 5 月 9 日

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW

计划入组 804 人 · 首次发布于 2016 年 2 月 2 日

Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity

计划入组 213 人 · 首次发布于 2017 年 1 月 6 日

Rare Genetic diseases therapeutics (Degron)

undisclosed program (rare genetic skin disease)

LUNAR-RARE2

LYSOSOMAL AND RARE DISORDERS RESEARCH AND TREATMENT CENTER, INC.

United States

Genetic Immunity

United States

Genetic Technologies Ltd

Matica Biotechnology Partners with Childs Cure to Develop Gene Therapy for Rare Genetic Disorders

Vosoritide Shows Promise for Rare Genetic Growth Disorders Beyond Achondroplasia in Phase 2 Trial

Five Major FDA Decisions Expected in November 2025 Across Cancer, Rare Disease, and Genetic Disorders