Phenotypes, Biomarkers and Pathophysiology in Spastic Ataxias

计划入组 250 人 · 首次发布于 2020 年 3 月 6 日

Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias

计划入组 84 人 · 首次发布于 2024 年 2 月 15 日

TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias

计划入组 4,000 人 · 首次发布于 2026 年 9 月 1 日

Undisclosed gene therapy (spinocerebellar ataxias)

AP4M1 gene therapy (AAV9, hereditary spastic paraplegia 50)

AAV9 vector-based AP4B1 gene therapy (hereditary spastic paraplegia 47)

FRIEDREICH'S ATAXIA RESEARCH ALLIANCE

United States

Alaxia SAS

France

Aplastic Anemia And Mds International Foundation Inc

United States

Digital Gait Analysis Shows Promise for Clinical Trials in SPG7

CD99L2 Identified as Novel Disease Gene for Spastic Ataxia and Hereditary Spastic Paraplegia

MRI Biomarkers Revolutionize Clinical Trial Design for Hereditary Ataxias