256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details


臨床試験数 : 180 薬物数 : 157 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134

  
1 trial found
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enrollment
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agemin
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PhaseCountries
1NCT02240407
(ClinicalTrials.gov)
October 17, 201711/9/2014Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe DiseaseEvaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)Pompe DiseaseGenetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical CreamUniversity of FloridaNULLActive, not recruiting18 Years50 YearsAll2Phase 1United States