Pompe's Disease

Also known as: Pompe Disease / Cardiac glycogenosis / Acid maltase deficiency / Glycogen storage disease type II / Type II glycogen storage disease

DrugDrug NameDrug Description
DB01272Alglucosidase alfaAn acid alpha- glucosidase (GAA) derivative used as an enzyme replacement therapy for the treatment of Pompe disease in infants and pediatric patients caused by GAA deficiency.
DB16099Avalglucosidase alfaA lysosomal glycogen-specific enzyme that is used to treat patients one year of age and older with late-onset Pompe disease, or lysosomal acid alpha-glucosidase (GAA) deficiency.
DrugDrug NamePhaseStatusCount
DB00545Pyridostigmine0Terminated1
DB00316Acetaminophen1Completed1
DB16099Avalglucosidase alfa1Completed1
DB01075Diphenhydramine1Completed1
DB00281Lidocaine1Completed1
DB00419Miglustat1Terminated1
DB00073Rituximab1Completed1
DB01001Salbutamol1Completed1
DB00877Sirolimus1Completed1
DB01272Alglucosidase alfa1 / 2Completed1
DB01272Alglucosidase alfa1 / 2Terminated1
DB16708Cipaglucosidase alfa1 / 2Active Not Recruiting1
DB01407Clenbuterol1 / 2Completed1
DB00419Miglustat1 / 2Active Not Recruiting1
DB01001Salbutamol1 / 2Completed1
DB05200AT22202Completed1
DB05200AT22202Terminated1
DB01272Alglucosidase alfa2Completed3
DB03206Duvoglustat2Completed1
DB03206Duvoglustat2Terminated1
DB01272Alglucosidase alfa4Completed4
DB01272Alglucosidase alfa4Recruiting1
DB01272Alglucosidase alfa4Terminated2
DB00188Bortezomib4Unknown Status1
DB00531Cyclophosphamide4Terminated1
DB00563Methotrexate4Completed1
DB00563Methotrexate4Terminated1
DB00563Methotrexate4Unknown Status1
DB00073Rituximab4Completed1
DB00073Rituximab4Terminated1
DB00073Rituximab4Unknown Status1
DB01001Salbutamol4Completed1
DB01272Alglucosidase alfaNot AvailableActive Not Recruiting1
DB01272Alglucosidase alfaNot AvailableCompleted2
DB01272Alglucosidase alfaNot AvailableUnknown Status1
DB16708Cipaglucosidase alfaNot AvailableAvailable1
DB00099FilgrastimNot AvailableRecruiting1
DB00419MiglustatNot AvailableCompleted1
DB00073RituximabNot AvailableCompleted1