A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
Status:
Completed
Trial end date:
2010-03-01
Target enrollment:
Participant gender:
Summary
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in
children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures
for HU treatment in Types II and III SMA; and to detect the clinical efficacy of HU treatment
in children with Types II and III SMA.