32. 自己貪食空胞性ミオパチー Autophagic vacuolar myopathy Clinical trials / Disease details


臨床試験数 : 1 薬物数 : 1 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

  
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agemin
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PhaseCountries
1NCT03882437
(ClinicalTrials.gov)
April 17, 201912/3/2019Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2BA Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With DDDanon DiseaseBiological: RP-A501Rocket Pharmaceuticals Inc.NULLRecruiting8 YearsN/AMale10Phase 1United States