Disease 指定難病
疾患数 : 338 - 臨床試験総数 : 34,520 / 薬物総数 : 19,957 - ( DrugBank : 2,195 ) / 標的遺伝子総数 : 613 - 標的パスウェイ総数 : 297
| 告示 番号 | 疾患名 ［疾患群］ | 臨床試験数 | Phase 1 / 2 / 3 / 4 | 薬物数 [ DrugBank ] | 標的遺伝子数 | パスウェイ数 | 国内患者数 医療費受給者証所持者数 (R4年度) |
|---|---|---|---|---|---|
| 84 | サルコイドーシス [呼] 💬 "Sarcoidosis" | 149 149 trials | 52 / 70 / 31 / 25 💬 | 202 202 drugs [ 78 78 drugs ] | 66 66 genes | 169 pathways | 15627 15,627人年齢分布  💬 |
| 85 | 特発性間質性肺炎 [呼] 💬 "Idiopathic interstitial pneumonia", "IIPs", "Idiopathic pulmonary fibrosis", "IPF", "Usual interstitial pneumonia", "UIP", "Non-specific interstitial pneumonia", "NSIP", "Acute interstitial pneumonia", "AIP", "Diffuse alveolar damage", "DAD", "Cryptogenic organizing pneumonia", "COP", "Organizing pneumonia", "OP", "Desquamative interstitial pneumonia", "DIP", "Respiratory bronchiolitis - associated interstitial lung disease", "RB-ILD", "Lymphocytic interstitial pneumonia", "LIP" | 627 627 trials | 216 / 257 / 167 / 59 💬 | 443 443 drugs [ 120 120 drugs ] | 99 99 genes | 212 pathways | 18399 18,399人年齢分布  💬 |
| 86 | 肺動脈性肺高血圧症 [呼] 💬 "Pulmonary arterial hypertension", "PAH", "IPAH", "HPAH", "Eisenmenger syndrome" | 1,205 1,205 trials | 404 / 309 / 485 / 170 💬 | 684 684 drugs [ 124 124 drugs ] | 100 100 genes | 193 pathways | 4529 4,529人年齢分布  💬 |
| 87 | 肺静脈閉塞症／肺毛細血管腫症 [呼] 💬 "Pulmonary veno-occlusive disease", "Pulmonary capillary hemangiomatosis", "PVOD", "PCH" | 3 3 trials | 1 / 1 / 0 / 0 💬 | 4 4 drugs [ 3 3 drugs ] | 4 4 genes | 45 pathways | 25 25人年齢分布  💬 |
| 88 | 慢性血栓塞栓性肺高血圧症 [呼] 💬 "Chronic thromboembolic pulmonary hypertension", "CTEPH", "Idiopathic chronic pulmonary thromboembolism" | 157 157 trials | 77 / 51 / 70 / 18 💬 | 107 107 drugs [ 22 22 drugs ] | 14 14 genes | 54 pathways | 5230 5,230人年齢分布  💬 |
| 89 | リンパ脈管筋腫症 [呼] 💬 "Lymphangioleiomyomatosis", "LAM", "Sporadic lymphangioleiomyomatosis", "Sporadic LAM", "Lymphangioleiomyomatosis with tuberous sclerosis", "TSC-LAM" | 38 38 trials | 14 / 18 / 8 / 2 💬 | 38 38 drugs [ 15 15 drugs ] | 18 18 genes | 118 pathways | 928 928人年齢分布  💬 |
| 228 | 閉塞性細気管支炎 [呼] 💬 "Bronchiolitis obliterans", "Obliterating bronchiolitis" | 97 97 trials | 49 / 40 / 36 / 16 💬 | 118 118 drugs [ 32 32 drugs ] | 33 33 genes | 156 pathways | 36 36人年齢分布  💬 |
| 229 | 肺胞蛋白症（自己免疫性又は先天性） [呼] 💬 "Autoimmune pulmonary alveolar proteinosis", "Congenital pulmonary alveolar proteinosis", "Hereditary pulmonary alveolar proteinosis", "Pulmonary alveolar proteinosis", "PAP", "Autoimmune PAP", "Congenital PAP" | 44 44 trials | 31 / 21 / 27 / 5 💬 | 22 22 drugs [ 7 7 drugs ] | 3 3 genes | 14 pathways | 238 238人年齢分布  💬 |
| 230 | 肺胞低換気症候群 [呼] 💬 "Alveolar hypoventilation syndrome", "AHS", "Hypoventilation syndrome", "Obesity hypoventilation syndrome", "OHS", "Congenital central hypoventilation syndrome", "CCHS", "Idiopathic central alveolar hypoventilation", "ICAH" | 6 6 trials | 0 / 2 / 2 / 0 💬 | 7 7 drugs [ 4 4 drugs ] | 16 16 genes | 26 pathways | 164 164人年齢分布  💬 |
| 231 | α1−アンチトリプシン欠乏症 [呼] 💬 "Alpha-1-antitrypsin deficiency", "AATD" | 93 93 trials | 47 / 59 / 29 / 8 💬 | 77 77 drugs [ 8 8 drugs ] | 10 10 genes | 36 pathways | 16 16人年齢分布  💬 |
| 277 | リンパ管腫症／ゴーハム病 [呼] 💬 "Lymphangiomatosis", "Generalized lymphatic anomaly", "Gorham disease", "Gorham-Stout disease", "Diffuse lymphangiomatosis", "Mass osteolysis" | 6 6 trials | 2 / 2 / 1 / 0 💬 | 2 2 drugs [ 2 2 drugs ] | 1 1 gene | 51 pathways | 74 74人年齢分布  💬 |
| 278 | 巨大リンパ管奇形（頚部顔面病変） [呼] 💬 "Huge lymphatic malformation with cervicofacial lesion", "Huge lymphatic malformation", "Lymphatic malformation" | 19 19 trials | 12 / 10 / 4 / 4 💬 | 22 22 drugs [ 7 7 drugs ] | 5 5 genes | 63 pathways | 24 24人年齢分布  💬 |
| 294 | 先天性横隔膜ヘルニア [呼] 💬 "Congenital diaphragmatic hernia" | 16 16 trials | 6 / 4 / 6 / 3 💬 | 26 26 drugs [ 5 5 drugs ] | 5 5 genes | 14 pathways | 12 12人年齢分布  💬 |
| 330 | 先天性気管狭窄症/先天性声門下狭窄症 [呼] 💬 [聴] 💬 "Congenital tracheal stenosis", "Congenital subglottic stenosis" | 1 1 trial | 0 / 1 / 0 / 0 💬 | 3 3 drugs [ 2 2 drugs ] | 0 - | 44 44人年齢分布  💬 |