Bmn 053    (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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1EUCTR2011-005042-35-GB
(EUCTR)
23/08/201318/03/2013A study to test if BMN 053 is safe and effective in people who suffer from Duchenne muscular dystrophyA Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: no
Male: yes
45Phase 1;Phase 2United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan