Disease The intractable diseases designated by MHLW, Japan
Diseases : 338 - Clinical trials : 33,695 / Drugs : 21,110 - ( DrugBank : 2,155 ) / Drug target genes : 623 - Drug target pathways : 291
Disease group: Respiratory diseases| ID | Disease name [Group] | Clinical trial Phase 1 / 2 / 3 / 4 | Drug [ DrugBank ] | Target gene Target pathway | Domestic patients (1) MHLW, (2) Med expenses recipients (FY2021) |
|---|---|---|---|---|---|
| 84 | Sarcoidosis [Resp] 💬 | 149 149 trials 53 / 64 / 30 / 26 💬 | 227 227 drugs [ 81 81 drugs ] | 82 82 genes 167 pathways | 15655 (1) 26,763 patients (2) 15,655 patients Age distribution  💬 |
| 85 | Idiopathic interstitial pneumonia [Resp] 💬 "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" | 598 598 trials 211 / 235 / 160 / 60 💬 | 435 435 drugs [ 116 116 drugs ] | 100 100 genes 210 pathways | 17665 (1) More than about 15,000 patients (Medical care eligibility certificate holders in fiscal year 2,014: 8,846 patients) (2) 17,665 patients Age distribution  💬 |
| 86 | Pulmonary arterial hypertension [Resp] 💬 "PAH", "IPAH", "HPAH", "Eisenmenger syndrome" | 1,181 1,181 trials 419 / 302 / 467 / 174 💬 | 701 701 drugs [ 126 126 drugs ] | 105 105 genes 192 pathways | 4319 (1) 2,299 patients (2) 4,319 patients Age distribution  💬 |
| 87 | Pulmonary veno-occlusive disease [Resp] 💬 "Pulmonary capillary hemangiomatosis", "PVOD", "PCH" | 3 3 trials 1 / 1 / 0 / 0 💬 | 4 4 drugs [ 3 3 drugs ] | 4 4 genes 45 pathways | 24 (1) About 100 patients (2) 24 patients Age distribution  💬 |
| 88 | Chronic thromboembolic pulmonary hypertension [Resp] 💬 "CTEPH", "Idiopathic chronic pulmonary thromboembolism" | 159 159 trials 82 / 51 / 70 / 19 💬 | 118 118 drugs [ 23 23 drugs ] | 13 13 genes 53 pathways | 4843 (1) 1,810 patients (2) 4,843 patients Age distribution  💬 |
| 89 | Lymphangioleiomyomatosis [Resp] 💬 "LAM", "Sporadic lymphangioleiomyomatosis", "Sporadic LAM", "Lymphangioleiomyomatosis with tuberous sclerosis", "TSC-LAM" | 39 39 trials 15 / 18 / 8 / 2 💬 | 46 46 drugs [ 20 20 drugs ] | 27 27 genes 137 pathways | 912 (1) 689 patients (2) 912 patients Age distribution  💬 |
| 228 | Bronchiolitis obliterans [Resp] 💬 "Obliterating bronchiolitis" | 96 96 trials 49 / 38 / 35 / 16 💬 | 125 125 drugs [ 34 34 drugs ] | 33 33 genes 155 pathways | 35 (1) About 300~500 patients (2) 35 patients Age distribution  💬 |
| 229 | Autoimmune pulmonary alveolar proteinosis [Resp] 💬 "Congenital pulmonary alveolar proteinosis", "Hereditary pulmonary alveolar proteinosis", "Pulmonary alveolar proteinosis", "PAP", "Autoimmune PAP", "Congenital PAP" | 43 43 trials 32 / 21 / 26 / 5 💬 | 34 34 drugs [ 8 8 drugs ] | 3 3 genes 14 pathways | 212 (1) About 900 patients (Autoimmune and congenital pulmonary alveolar proteinoses) (2) 212 patients Age distribution  💬 |
| 230 | Alveolar hypoventilation syndrome [Resp] 💬 "AHS", "Hypoventilation syndrome", "Obesity hypoventilation syndrome", "OHS", "Congenital central hypoventilation syndrome", "CCHS", "Idiopathic central alveolar hypoventilation", "ICAH" | 8 8 trials 0 / 2 / 2 / 0 💬 | 11 11 drugs [ 5 5 drugs ] | 18 18 genes 28 pathways | 145 (1) About 3,000 patients (2) 145 patients Age distribution  💬 |
| 231 | Alpha-1-antitrypsin deficiency [Resp] 💬 "AATD" | 89 89 trials 43 / 58 / 27 / 8 💬 | 89 89 drugs [ 16 16 drugs ] | 35 35 genes 47 pathways | 16 (1) Less than 100 patients (2) 16 patients Age distribution  💬 |
| 277 | Lymphangiomatosis [Resp] 💬 "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 | 56 (1) About 100 patients (estimated by the national survey of the research group) (2) 56 patients Age distribution  💬 |
| 278 | Huge lymphatic malformation with cervicofacial lesion [Resp] 💬 "Huge lymphatic malformation", "Lymphatic malformation" | 22 22 trials 13 / 13 / 4 / 4 💬 | 27 27 drugs [ 7 7 drugs ] | 5 5 genes 63 pathways | 17 (1) About 600 patients (2) 17 patients Age distribution  💬 |
| 294 | Congenital diaphragmatic hernia [Resp] 💬 | 15 15 trials 6 / 2 / 7 / 3 💬 | 36 36 drugs [ 6 6 drugs ] | 5 5 genes 14 pathways | 13 (1) About 5,000 patients (2) 13 patients Age distribution  💬 |
| 330 | Congenital tracheal stenosis [Resp] 💬 "Congenital subglottic stenosis" | 1 1 trial 0 / 1 / 0 / 0 💬 | 3 3 drugs [ 2 2 drugs ] | 0 - | 41 (1) About 1,000 patients (2) 41 patients Age distribution  💬 |