Mitochondrial Dysfunction in Phelan-McDermid Syndrome

计划入组 51 人 · 首次发布于 2013 年 12 月 3 日

Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

计划入组 18 人 · 首次发布于 2016 年 3 月 16 日

Mapping the Phenotype in Adults With Phelan-McDermid Syndrome

计划入组 24 人 · 首次发布于 2018 年 2 月 8 日

Phelan-McDermid Syndrome(Ksilink)

SHANK3 gene therapy (Phelan McDermid syndrome)

SHANK3 gene therapy (AAV, Phelan McDermid syndrome)

ALPORT SYNDROME FOUNDATION

United States

International Rett Syndrome Foundation

United States

RETT SYNDROME RESEARCH TRUST

United States

FDA Grants Orphan Drug Designation to NeuroNOS for Phelan-McDermid Syndrome Treatment

Neuren Pharmaceuticals Launches First-Ever Phase 3 Trial for Phelan-McDermid Syndrome

Phelan-McDermid Syndrome Prevalence Estimated at 1 in 7,300, Far Higher Than Previously Thought