DDrare: Database of Drug Development for Rare Diseases

ID	Disease name (Link within this page)	Number of trials
2	Amyotrophic lateral sclerosis	0
25	Progressive multifocal leukoencephalopathy	0
93	Primary biliary cholangitis [Primary biliary cirrhosis (~Mar 2017)]	0
265	Lipodystrophy	0
325	Hereditary autoinflammatory syndrome	0

2. Amyotrophic lateral sclerosis [ 508 clinical trials, 530 drugs, (DrugBank: 146 drugs), 170 drug target genes, 221 drug target pathways]

Searched query = "Amyotrophic lateral sclerosis", "ALS"
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.

25. Progressive multifocal leukoencephalopathy [ 23 clinical trials, 33 drugs, (DrugBank: 20 drugs), 7 drug target genes, 36 drug target pathways]

Searched query = "Progressive multifocal leukoencephalopathy", "PML", "Leukoencephalopathy, progressive multifocal"
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.

265. Lipodystrophy [ 109 clinical trials, 164 drugs, (DrugBank: 59 drugs), 26 drug target genes, 94 drug target pathways]

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons 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.

325. Hereditary autoinflammatory syndrome [ 6 clinical trials, 10 drugs, (DrugBank: 6 drugs), 2 drug target genes, 33 drug target pathways]

Searched query = "Hereditary autoinflammatory syndrome", "NLRC4 abnormality", "Adenosine deaminase 2 deficiency", "ADA2 deficiency", "Aicardi-Goutieres syndrome", "A20 haploinsufficiency"
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.