Studies Of Hereditary Neurological Disease: Clinical Trials

Neurological Disorders And Stroke

Investigators

Linked publications & trials

Abstract

The purpose of this research program is to develop safe and effective treatments for hereditary neurological disorders. Specific research accomplishments in the past year include the following: (1) a collaborative study to establish reliable biomarkers for spinal muscular atrophy, (2) completion of a phase 2 study of high dose idebenone treatment for Friedreich's ataxia, and (3) development of a phase 2 clinical trial to test the feasibility of dutasteride treatment for spinal and bulbar muscular atrophy (Kennedy's disease).