Disease The intractable diseases designated by MHLW, Japan
Diseases : 338 - Clinical trials : 34,520 / Drugs : 19,957 - ( DrugBank : 2,195 ) / Drug target genes : 613 - Drug target pathways : 297
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 52 / 70 / 31 / 25 💬 | 202 202 drugs [ 78 78 drugs ] | 66 66 genes 169 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" | 627 627 trials 216 / 257 / 167 / 59 💬 | 443 443 drugs [ 120 120 drugs ] | 99 99 genes 212 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" | 1,205 1,205 trials 404 / 309 / 485 / 170 💬 | 684 684 drugs [ 124 124 drugs ] | 100 100 genes 193 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" | 157 157 trials 77 / 51 / 70 / 18 💬 | 107 107 drugs [ 22 22 drugs ] | 14 14 genes 54 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" | 38 38 trials 14 / 18 / 8 / 2 💬 | 38 38 drugs [ 15 15 drugs ] | 18 18 genes 118 pathways | 912 (1) 689 patients (2) 912 patients Age distribution  💬 |
| 228 | Bronchiolitis obliterans [Resp] 💬 "Obliterating bronchiolitis" | 97 97 trials 49 / 40 / 36 / 16 💬 | 118 118 drugs [ 32 32 drugs ] | 33 33 genes 156 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" | 44 44 trials 31 / 21 / 27 / 5 💬 | 22 22 drugs [ 7 7 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" | 6 6 trials 0 / 2 / 2 / 0 💬 | 7 7 drugs [ 4 4 drugs ] | 16 16 genes 26 pathways | 145 (1) About 3,000 patients (2) 145 patients Age distribution  💬 |
| 231 | Alpha-1-antitrypsin deficiency [Resp] 💬 "AATD" | 93 93 trials 47 / 59 / 29 / 8 💬 | 77 77 drugs [ 8 8 drugs ] | 10 10 genes 36 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" | 19 19 trials 12 / 10 / 4 / 4 💬 | 22 22 drugs [ 7 7 drugs ] | 5 5 genes 63 pathways | 17 (1) About 600 patients (2) 17 patients Age distribution  💬 |
| 294 | Congenital diaphragmatic hernia [Resp] 💬 | 16 16 trials 6 / 4 / 6 / 3 💬 | 26 26 drugs [ 5 5 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  💬 |