DDrare: Database of Drug Development for Rare Diseases

Sapropterin (kuvan) (DrugBank: Sapropterin)

5 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
51	全身性強皮症	0
86	肺動脈性肺高血圧症	0
124	皮質下梗塞と白質脳症を伴う常染色体優性脳動脈症	0
240	フェニルケトン尿症	1
299	嚢胞性線維症	0

51. 全身性強皮症　［臨床試験数：466，薬物数：536（DrugBank：142），標的遺伝子数：110，標的パスウェイ数：210］

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. 肺動脈性肺高血圧症　［臨床試験数：1,083，薬物数：667（DrugBank：122），標的遺伝子数：98，標的パスウェイ数：185］

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. 皮質下梗塞と白質脳症を伴う常染色体優性脳動脈症　［臨床試験数：11，薬物数：13（DrugBank：4），標的遺伝子数：3，標的パスウェイ数：11］

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. フェニルケトン尿症　［臨床試験数：125，薬物数：95（DrugBank：11），標的遺伝子数：1，標的パスウェイ数：5］

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. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］

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