Pompe's Disease

Also known as: Acid Maltase Deficiency / Pompe Disease / Acid Maltase Deficiency (AMD) / Pompe-Disease / Glycogen Storage Disease Type II / Glycogen Storage Disease Type II (GSD-II) / Glycogen Storage Disease Type II GSD II / Glycogen Storage Disease Type II (GSD II) / Glycogen Storage Disease Type II;Pompe's Disease / Glycogen Storage Disease Type II-Pompe's Disease / Glycogen Storage Disease Type II Pompe Disease / Cardiac glycogenosis (disorder) / Glycogen storage disease, type II (disorder) / Type II glycogen storage disease / Deficiency of alpha-glucosidase (disorder)

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
DB17658ACTUS-1011 / 2Active Not Recruiting1
DB01272Alglucosidase alfa1 / 2Completed1
DB01272Alglucosidase alfa1 / 2Terminated1
DB16708Cipaglucosidase alfa1 / 2Active Not Recruiting1
DB01407Clenbuterol1 / 2Completed1
DB17623Clervonafusp alfa1 / 2Terminated1
DB00419Miglustat1 / 2Active Not Recruiting1
DB17665Reveglucosidase alfa1 / 2Completed1
DB01001Salbutamol1 / 2Completed1
DB05200AT22202Completed1
DB05200AT22202Terminated1
DB01272Alglucosidase alfa2Active Not Recruiting1
DB01272Alglucosidase alfa2Completed6
DB16099Avalglucosidase alfa2Active Not Recruiting1
DB16099Avalglucosidase alfa2Completed1
DB03206Duvoglustat2Completed1
DB03206Duvoglustat2Terminated1
DB17665Reveglucosidase alfa2Terminated1
DB01272Alglucosidase alfa2 / 3Completed2
DB01272Alglucosidase alfa3Completed3
DB16099Avalglucosidase alfa3Completed1
DB16099Avalglucosidase alfa3Recruiting1
DB01272Alglucosidase alfa4Active Not Recruiting1
DB01272Alglucosidase alfa4Completed7
DB01272Alglucosidase alfa4Terminated3
DB16099Avalglucosidase alfa4Active Not Recruiting1
DB00188Bortezomib4Unknown Status1
DB00531Cyclophosphamide4Terminated1
DB00563Methotrexate4Completed1
DB00563Methotrexate4Terminated1
DB00563Methotrexate4Unknown Status1
DB00073Rituximab4Completed1
DB00073Rituximab4Terminated1
DB00073Rituximab4Unknown Status1
DB01001Salbutamol4Completed1
DB01272Alglucosidase alfaNot AvailableActive Not Recruiting1
DB01272Alglucosidase alfaNot AvailableApproved for Marketing2
DB01272Alglucosidase alfaNot AvailableCompleted3
DB01272Alglucosidase alfaNot AvailableNot Yet Recruiting1
DB01272Alglucosidase alfaNot AvailableRecruiting2
DB01272Alglucosidase alfaNot AvailableUnknown Status1
DB16099Avalglucosidase alfaNot AvailableRecruiting2
DB16708Cipaglucosidase alfaNot AvailableAvailable1
DB16708Cipaglucosidase alfaNot AvailableRecruiting1
DB00099FilgrastimNot AvailableRecruiting1
DB00419MiglustatNot AvailableAvailable1
DB00419MiglustatNot AvailableCompleted1
DB00419MiglustatNot AvailableRecruiting1
DB00073RituximabNot AvailableCompleted1