Department of Neurology

Pediatric Motor Disorders Research Program

Ongoing or Completed Trials & Studies

Enrolling/Ongoing: 

Clinical and Genetic Studies in Spinal Muscular Atrophy

Metabolic Dysfunction in SMA: impact of nutritional management

Prospective Study of Bone Abnormalities in SMA

STOP SMA: Phenylbutyrate trial in pre-symptomatic infants with SMA 

CARNI-VAL SMA Type I (multi-center) see www.projectcuresma.org

Study of Pediatric Motor Disorders 

Completed: 

Motor Unit Number Estimation in SMA

Pilot Trial of Valproic Acid in Spinal Muscular Atrophy

Pilot Trial of Sodium Phenylbutyrate in Spinal Muscular Atrophy

CARNI-VAL SMA TRIAL (multi-center) see www.projectcuresma.org