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
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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