DDrare: Database of Drug Development for Rare Diseases

Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhgaa]) (DrugBank: Alglucosidase alfa, Alpha-glucosidase)

1 disease

ID	Disease name (Link within this page)	Number of trials
256	Muscle glycogenosis	1

256. Muscle glycogenosis [ 161 clinical trials, 114 drugs, (DrugBank: 23 drugs), 26 drug target genes, 106 drug target pathways]

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 161 trial found

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00520143 (ClinicalTrials.gov)	July 2007	21/8/2007	Alglucosidase Alfa Temporary Access Program	Alglucosidase Alfa Temporary Access Program	Glycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-Onset);Acid Maltase Deficiency Disease;Glycogenosis 2	Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])	Genzyme, a Sanofi Company	NULL	Approved for marketing	18 Years	N/A	Both		N/A	United States