Pompe Disease (Late-Onset)

Also known as: Late Onset Pompe Disease / Late-onset Pompe Disease / Pompe Disease Late-Onset

Drug	Drug Name	Drug Description
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.
DB16708	Cipaglucosidase alfa	A recombinant human acid alpha-glucosidase used to treat late-onset Pompe disease in adults.

Drug	Drug Name	Target	Type
DB16099	Avalglucosidase alfa	Cation-independent mannose-6-phosphate receptor	target
DB16708	Cipaglucosidase alfa	Cation-independent mannose-6-phosphate receptor	target
DB16708	Cipaglucosidase alfa	Alpha glucosidase (GAA)	target

Drug	Drug Name	Phase	Status	Count
DB01272	Alglucosidase alfa	2	Completed	2
DB01407	Clenbuterol	2	Withdrawn	1
DB01272	Alglucosidase alfa	3	Completed	3
DB16708	Cipaglucosidase alfa	3	Active Not Recruiting	1
DB16708	Cipaglucosidase alfa	3	Completed	1
DB16708	Cipaglucosidase alfa	3	Recruiting	1
DB00419	Miglustat	3	Active Not Recruiting	1
DB00419	Miglustat	3	Completed	1
DB00419	Miglustat	3	Recruiting	1
DB17665	Reveglucosidase alfa	3	Terminated	1
DB01272	Alglucosidase alfa	4	Completed	2
DB01272	Alglucosidase alfa	Not Available	Approved for Marketing	1
DB01272	Alglucosidase alfa	Not Available	Unknown Status	1