Studies Of Hereditary Neurological Disease: Clinical Trials

National Institute Of Neurological Disorders And Stroke

Investigators

Linked publications, trials & patents

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) completion of a phase 2 study of high dose idebenone treatment for Friedreich's ataxia, and (3) implementation of a phase 2 clinical trial to test the feasibility of dutasteride treatment for spinal and bulbar muscular atrophy (Kennedy's disease). The idebenone trial showed evidence of neurological efficacy with intermediate and high dose treatment in ambulatory subjects. Analysis of other outcome measures is in progress.