DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
2	筋萎縮性側索硬化症	0
25	進行性多巣性白質脳症	0
93	原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］	0
265	脂肪萎縮症	0
325	遺伝性自己炎症疾患	0

2. 筋萎縮性側索硬化症　［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］

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. 進行性多巣性白質脳症　［臨床試験数：23，薬物数：33（DrugBank：20），標的遺伝子数：7，標的パスウェイ数：36］

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. 原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］　［臨床試験数：230，薬物数：215（DrugBank：51），標的遺伝子数：34，標的パスウェイ数：107］

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. 脂肪萎縮症　［臨床試験数：109，薬物数：164（DrugBank：59），標的遺伝子数：26，標的パスウェイ数：94］

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. 遺伝性自己炎症疾患　［臨床試験数：6，薬物数：10（DrugBank：6），標的遺伝子数：2，標的パスウェイ数：33］

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.