Overview

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route o

Status:
Terminated
Trial end date:
2016-09-01
Target enrollment:
Participant gender:
Summary
The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
Phase:
Phase 2
Details
Lead Sponsor:
BioMarin Pharmaceutical