A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
Status:
Completed
Trial end date:
2012-02-01
Target enrollment:
Participant gender:
Summary
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in
children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU
treatment in Type I SMA; and to detect the clinical efficacy of HU treatment in children with
Type I SMA.