Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

首次发布于 2014 年 11 月 25 日

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

计划入组 28 人 · 首次发布于 2014 年 6 月 11 日

Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)

首次发布于 2011 年 12 月 20 日

AAV2 smCBA based ND4 gene therapy (Leber hereditary optic neuropathy)

Idebenone(Raxone)

C₁₉H₃₀O₅ · Rx

RNA actuator therapeutic for hereditary angioedema(CAMP4 Therapeutics)

Orthocell Ltd

Biora Therapeutics

United States

NeuroBo Pharmaceuticals

United States

Subthreshold Electrical Stimulation Shows Promise in LHON Treatment

Rare Disease Clinical Trials Face Logistical Challenges: Experts to Address Solutions at Brussels Conference

NICE Recommends First NHS Treatment for Rare Eye Disease LHON, Ending Access Disparity in England