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An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
Sponsor: BioMarin Pharmaceutical
Summary
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.
Official title: An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Key Details
Gender
MALE
Age Range
4 Years - Any
Study Type
INTERVENTIONAL
Enrollment
18
Start Date
2026-04-20
Completion Date
2031-12
Last Updated
2026-07-24
Healthy Volunteers
No
Conditions
Interventions
BMN 351
Anti-sense Oligonucleotide BMN 351 will be administered intravenously
Locations (6)
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy, Italy
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy, Italy
Leids Universitair Medisch Centrum
Leiden, Netherlands, Netherlands
Hospital Viamed Santa Angela De la Cruz
Seville, Spain, Spain
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey, Turkey (Türkiye)
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom, United Kingdom