DDrare: Database of Drug Development for Rare Diseases

ID	Disease name (Link within this page)	Number of trials
20	Adrenoleukodystrophy	0
93	Primary biliary cholangitis [Primary biliary cirrhosis (~Mar 2017)]	0
94	Primary sclerosing cholangitis	0
160	Congenital ichthyosis	0
316	Carnitine cycle disorder	0
324	Methylglutaconic aciduria	0

20. Adrenoleukodystrophy [ 49 clinical trials, 86 drugs, (DrugBank: 29 drugs), 18 drug target genes, 112 drug target pathways]

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
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.

93. Primary biliary cholangitis [Primary biliary cirrhosis (~Mar 2017)] [ 230 clinical trials, 215 drugs, (DrugBank: 51 drugs), 34 drug target genes, 107 drug target pathways]

Searched query = "Primary biliary cholangitis", "Primary biliary cirrhosis", "PBC"
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.

94. Primary sclerosing cholangitis [ 134 clinical trials, 105 drugs, (DrugBank: 37 drugs), 18 drug target genes, 131 drug target pathways]

Searched query = "Primary sclerosing cholangitis", "PSC"
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.

160. Congenital ichthyosis [ 32 clinical trials, 62 drugs, (DrugBank: 18 drugs), 15 drug target genes, 107 drug target pathways]

Searched query = "Congenital ichthyosis", "Keratinopathic ichthyosis", "Epidermolytic ichthyosis", "Harlequin ichthyosis", "Congenital Ichthyosiform Erythroderma", "Foliate ichthyosis", "Ichthyosis syndrome", "Netherton syndrome", "Sjogren-Larsson syndrome", "Sjögren-Larsson syndrome", "Keratitis-ichtyosis-deafness syndrome", "Dorfman-Chanarin syndrome", "Neutral lipid storage disease", "NLSD", "Multiple sulfatase deficiency", "Austin disease", "Recessive X-linked ichthyosis", "RXLI", "X-linked recessive ichthyosis", "Ichthyosis, brittle hair, impaired intelligence, decreased fertility and short stature", "IBID", "Trichothiodystrophy", "Follicular ichthyosis", "Congenital hemidysplasia, ichthyosiform erythroderma or nevus, and limb defects syndrome", "CHILD syndrome", "Conradi-Hunermann-Happle syndrome", "Conradi-Hünermann-Happle syndrome", "CHHS"
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.

316. Carnitine cycle disorder [ 4 clinical trials, 10 drugs, (DrugBank: 4 drugs), 2 drug target genes, 9 drug target pathways]

Searched query = "Carnitine cycle disorder", "Carnitine palmitoyltransferase I deficiency", "CPT1 deficiency", "Carnitine palmitoyltransferase II deficiency", "CPT2 deficiency", "Carnitine-acylcarnitine translocase deficiency", "CACT deficiency", "Carnitine transporter deficiency", "OCTN-2 deficiency"
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.

324. Methylglutaconic aciduria [ 3 clinical trials, 4 drugs, (DrugBank: 3 drugs), 1 drug target gene, 8 drug target pathways]

Searched query = "Methylglutaconic aciduria", "3-methylglutaconyl-CoA hydratase deficiency", "3-MGA", "Barth syndrome", "Costeff syndrome", "Mitochondrial respiratory chain disorder", "Dilated cardiomyopathy with ataxia syndrome", "DCMA 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.