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 32. 自己貪食空胞性ミオパチー [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] 

Searched query = "Autophagic vacuolar myopathy", "Danon disease", "X-linked myopathy with excessive autophagy", "XMEA"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03882437April 17, 201915 July 2019Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2BGene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of 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 Danon DiseaseDanon DiseaseBiological: RP-A501Rocket Pharmaceuticals Inc.Recruiting8 YearsN/AMale24Phase 1United States