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 alfaAglucosidase alfa consists of the human enzyme acid alpha-glucosidase (GAA) which is essential for the degradation of glygogen to glucose in lysosomes. It is encoded by the most predominant of nine observed haplotypes of this gene. Aglucosidase alfa is produced by recombinant DNA technology in a Chinese hamster ovary cell line. Alglucosidase alfa degrades glycogen by catalyzing the hydrolysis of a-1,4- and a-1,6- glycosidic linkages of lysosomal glycogen. Structurally, Alglucosidase alfa is a glycoprotein with a calculated mass of 98,008 daltons for the 883 residue mature polypeptide chain, and a total mass of approximately 109,000 daltons, including carbohydrates. It is used for the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
DrugDrug NamePhaseStatusCount
DB00545Pyridostigmine0Terminated1
DB00316Acetaminophen1Recruiting1
DB01075Diphenhydramine1Recruiting1
DB09462Glycerin1Recruiting1
DB01202Levetiracetam1Recruiting1
DB00281Lidocaine1Recruiting1
DB00419Miglustat1Terminated1
DB00073Rituximab1Recruiting1
DB01001Salbutamol1Completed1
DB00877Sirolimus1Recruiting1
DB08604Triclosan1Recruiting1
DB01593Zinc1Recruiting1
DB01272Alglucosidase alfa1 / 2Active Not Recruiting1
DB01272Alglucosidase alfa1 / 2Completed1
DB01407Clenbuterol1 / 2Completed1
DB00419Miglustat1 / 2Recruiting1
DB01001Salbutamol1 / 2Completed1
DB05200AT22202Completed1
DB05200AT22202Terminated1
DB01272Alglucosidase alfa2Completed2
DB03206Duvoglustat2Completed1
DB03206Duvoglustat2Terminated1
DB00334Olanzapine2Completed1
DB01272Alglucosidase alfa4Active Not Recruiting2
DB01272Alglucosidase alfa4Completed2
DB01272Alglucosidase alfa4Terminated1
DB00188Bortezomib4Recruiting1
DB00531Cyclophosphamide4Active Not Recruiting1
DB00563Methotrexate4Active Not Recruiting1
DB00563Methotrexate4Completed1
DB00563Methotrexate4Recruiting1
DB00073Rituximab4Active Not Recruiting1
DB00073Rituximab4Completed1
DB00073Rituximab4Recruiting1
DB01001Salbutamol4Completed1
DB01272Alglucosidase alfaNot AvailableEnrolling by Invitation1
DB01272Alglucosidase alfaNot AvailableNot Yet Recruiting1
DB01272Alglucosidase alfaNot AvailableRecruiting2
DB00419MiglustatNot AvailableCompleted1
DB00073RituximabNot AvailableCompleted1