DDrare: Database of Drug Development for Rare Diseases

ID	Disease name (Link within this page)	Number of trials
5	Progressive supranuclear palsy	0
6	Parkinson disease	0
7	Corticobasal degeneration	0
19	Lysosomal storage disease	0
96	Crohn disease	0
97	Ulcerative colitis	0
127	Frontotemporal lobar degeneration	0
283	Acquired pure red cell aplasia	0
284	Diamond-Blackfan anemia	0
299	Cystic fibrosis	0

5. Progressive supranuclear palsy [ 82 clinical trials, 107 drugs, (DrugBank: 36 drugs), 60 drug target genes, 90 drug target pathways]

Searched query = "Progressive supranuclear palsy", "PSP"
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.

6. Parkinson disease [ 2,123 clinical trials, 2,046 drugs, (DrugBank: 324 drugs), 183 drug target genes, 198 drug target pathways]

Searched query = "Parkinson 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.

7. Corticobasal degeneration [ 16 clinical trials, 33 drugs, (DrugBank: 11 drugs), 6 drug target genes, 13 drug target pathways]

Searched query = "Corticobasal degeneration", "Corticobasal syndrome", "CBD"
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.

19. Lysosomal storage disease [ 784 clinical trials, 673 drugs, (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways]

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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.

96. Crohn disease [ 2,209 clinical trials, 1,276 drugs, (DrugBank: 240 drugs), 166 drug target genes, 210 drug target pathways]

Searched query = "Crohn disease", "Terminal ileitis"
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.

97. Ulcerative colitis [ 2,269 clinical trials, 1,331 drugs, (DrugBank: 241 drugs), 114 drug target genes, 181 drug target pathways]

Searched query = "Ulcerative colitis"
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.

127. Frontotemporal lobar degeneration [ 74 clinical trials, 83 drugs, (DrugBank: 28 drugs), 29 drug target genes, 99 drug target pathways]

Searched query = "Frontotemporal lobar degeneration", "Frontotemporal dementia", "Semantic dementia"
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.

283. Acquired pure red cell aplasia [ 17 clinical trials, 36 drugs, (DrugBank: 19 drugs), 16 drug target genes, 91 drug target pathways]

Searched query = "Acquired pure red cell aplasia", "Pure red cell aplasia"
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.

284. Diamond-Blackfan anemia [ 37 clinical trials, 110 drugs, (DrugBank: 34 drugs), 22 drug target genes, 121 drug target pathways]

Searched query = "Diamond-Blackfan anemia"
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.

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.