DDrare: Database of Drug Development for Rare Diseases

Sapropterin (kuvan) (DrugBank: Sapropterin)

5 diseases

ID	Disease name (Link within this page)	Number of trials
51	Scleroderma	0
86	Pulmonary arterial hypertension	0
124	Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy	0
240	Phenylketonuria	1
299	Cystic fibrosis	0

51. Scleroderma [ 466 clinical trials, 536 drugs, (DrugBank: 142 drugs), 110 drug target genes, 210 drug target pathways]

Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 466 trial found

86. Pulmonary arterial hypertension [ 1,083 clinical trials, 667 drugs, (DrugBank: 122 drugs), 98 drug target genes, 185 drug target pathways]

Searched query = "Pulmonary arterial hypertension", "PAH", "IPAH", "HPAH", "Eisenmenger syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 1,083 trial found

124. Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy [ 11 clinical trials, 13 drugs, (DrugBank: 4 drugs), 3 drug target genes, 11 drug target pathways]

Searched query = "Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy", "CADASIL", "Autosomal dominant cerebral artery disease with subcortical infarct and leukoencephalopathy", "Autosomal dominant cerebral artery disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 11 trial found

240. Phenylketonuria [ 125 clinical trials, 95 drugs, (DrugBank: 11 drugs), 1 drug target gene, 5 drug target pathways]

Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
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 / 125 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	NCT00730080 (ClinicalTrials.gov)	July 2008	1/8/2008	Sapropterin in Individuals With Phenylketonuria	Effects of Sapropterin on Brain and Cognition in Individuals With Phenylketonuria	Phenylketonuria	Drug: Sapropterin (Kuvan)	Washington University School of Medicine	BioMarin Pharmaceutical;University of Missouri-Columbia	Completed	6 Years	50 Years	All	45		United States

299. Cystic fibrosis [ 1,592 clinical trials, 1,539 drugs, (DrugBank: 255 drugs), 81 drug target genes, 162 drug target pathways]

Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 1,592 trial found