Pompe's Disease

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

Drug	Drug Name	Drug Description
DB01272	Alglucosidase alfa	An 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.
DB16099	Avalglucosidase alfa	A 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.

Drug	Drug Name	Target	Type
DB01272	Alglucosidase alfa	Cation-dependent mannose-6-phosphate receptor	target
DB01272	Alglucosidase alfa	Glycogen	target
DB16099	Avalglucosidase alfa	Cation-independent mannose-6-phosphate receptor	target

Drug	Drug Name	Phase	Status	Count
DB00545	Pyridostigmine	0	Terminated	1
DB00316	Acetaminophen	1	Completed	1
DB16099	Avalglucosidase alfa	1	Completed	1
DB01075	Diphenhydramine	1	Completed	1
DB00281	Lidocaine	1	Completed	1
DB00419	Miglustat	1	Terminated	1
DB00073	Rituximab	1	Completed	1
DB01001	Salbutamol	1	Completed	1
DB00877	Sirolimus	1	Completed	1
DB01272	Alglucosidase alfa	1 / 2	Completed	1
DB01272	Alglucosidase alfa	1 / 2	Terminated	1
DB16708	Cipaglucosidase alfa	1 / 2	Active Not Recruiting	1
DB01407	Clenbuterol	1 / 2	Completed	1
DB00419	Miglustat	1 / 2	Active Not Recruiting	1
DB01001	Salbutamol	1 / 2	Completed	1
DB05200	AT2220	2	Completed	1
DB05200	AT2220	2	Terminated	1
DB01272	Alglucosidase alfa	2	Completed	3
DB03206	Duvoglustat	2	Completed	1
DB03206	Duvoglustat	2	Terminated	1
DB01272	Alglucosidase alfa	4	Completed	4
DB01272	Alglucosidase alfa	4	Recruiting	1
DB01272	Alglucosidase alfa	4	Terminated	2
DB00188	Bortezomib	4	Unknown Status	1
DB00531	Cyclophosphamide	4	Terminated	1
DB00563	Methotrexate	4	Completed	1
DB00563	Methotrexate	4	Terminated	1
DB00563	Methotrexate	4	Unknown Status	1
DB00073	Rituximab	4	Completed	1
DB00073	Rituximab	4	Terminated	1
DB00073	Rituximab	4	Unknown Status	1
DB01001	Salbutamol	4	Completed	1
DB01272	Alglucosidase alfa	Not Available	Active Not Recruiting	1
DB01272	Alglucosidase alfa	Not Available	Completed	2
DB01272	Alglucosidase alfa	Not Available	Unknown Status	1
DB16708	Cipaglucosidase alfa	Not Available	Available	1
DB00099	Filgrastim	Not Available	Recruiting	1
DB00419	Miglustat	Not Available	Completed	1
DB00073	Rituximab	Not Available	Completed	1