Overview

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Status:
RECRUITING
Trial end date:
2026-09-30
Target enrollment:
Participant gender:
Summary
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
Phase:
PHASE1
Details
Lead Sponsor:
BioMarin Pharmaceutical