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. We recently reported improvement with functional exercise in a patient with spinal and bulbar muscular atrophy (SBMA). Natural history studies are currently underway for SBMA and ALS4, an early-onset hereditary motor neuron disease caused by mutations in senataxin, and one or more interventional studies for SBMA are under consideration for next year. The interventional studies will be designed based on muscle function and muscle imaging data collected in our SBMA natural history study and previous clinical studies by our group and others.