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Bmn 053    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
113Muscular dystrophy1

113. Muscular dystrophy    [ 567 clinical trials,   442 drugs,   (DrugBank: 93 drugs),   55 drug target genes,   151 drug target pathways]
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
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
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