Regimen selection phase group 2    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
113筋ジストロフィー1

113. 筋ジストロフィー [臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151
Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 567 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