113. Muscular dystrophy Clinical trials / Disease details


Clinical trials : 622 Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168

  
1 trial found
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1NCT01957059
(ClinicalTrials.gov)
June 20132/7/2013A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.Duchenne Muscular DystrophyDrug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing ExtensionBioMarin PharmaceuticalNULLTerminated5 Years18 YearsMale9Phase 1/Phase 2Belgium;France;Italy;Netherlands;United Kingdom