Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

首次发布于 2011 年 10 月 28 日

Inherited Reproductive Disorders

计划入组 850 人 · 首次发布于 2011 年 12 月 28 日

National Registry for Ichthyosis and Related Disorders

计划入组 610 人 · 首次发布于 2003 年 12 月 19 日

inherited macular degeneration program

GPR139 antagonists (CNS disorders/motor disorders)

ABCA4 gene therapy (AAV, inherited retinal dystrophy)

The Center For Comprehensive Care and Diagnosis of Inherited Blood Disorders

United States

Institute For Neurodegenerative Disorders

United States

Ascendis Pharma Growth Disorders A/S

Denmark

In Utero Gene Therapy Approaches First FDA Trial After 25 Years of Development

PYC Therapeutics Advances Two RNA-Based Therapies for Inherited Blindness Conditions

Real-World Gene Therapy Implementation Shows Promise for Blood Disorders Despite Timeline Challenges