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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2011-005040-10-GB
(EUCTR)
22/04/201310/10/2012A study to test if multiple injections of BMN 045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophyA phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by BMN 045-induced DMD exon 45 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]
Product Name: BMN 045
Product Code: BMN 045
INN or Proposed INN: PS220
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
45Phase 1;Phase 2France;Belgium;Italy;United Kingdom