Finding studies
Finding studies
Take this into the appointment.
Saves the questions and what to expect into your notes, next to the visit they belong to.
Kristy Riddle, RN, BSN
CONTACT
Ben Eckert, BA
CONTACT
Lead
Berge Minassian
With
A Phase 1/2 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease A single cohort will be evaluated in the study: N=10 • Initial dose of 15 mg ION283 intrathecal bolus (ITB) injection every 12 weeks. The study consists of 2 periods: * Screening Period: 4 weeks * Open label Treatment Period: 24 months
Age
10–18
Sex
ALL
Healthy volunteers
Not accepted