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Scaav9.u7.acca    (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
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT04240314
(ClinicalTrials.gov)		January 15, 202022/1/2020AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: scAAV9.U7.ACCAMegan WaldropAudentes TherapeuticsEnrolling by invitation6 Months13 YearsMale3Phase 1;Phase 2United States