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 trials 
 52 / 70 / 31 / 25 💬 
 202 drugs 
 [ 78 drugs
 66 genes 
 169 pathways 
(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 trials 
 216 / 257 / 167 / 59 💬 
 443 drugs 
 [ 120 drugs
 99 genes 
 212 pathways 
(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 trials 
 404 / 309 / 485 / 170 💬 
 684 drugs 
 [ 124 drugs
 100 genes 
 193 pathways 
(1) 2,299 patients
(2) 4,319 patients
Age distribution💬
 87 Pulmonary veno-occlusive disease [Resp] 💬
"Pulmonary capillary hemangiomatosis", "PVOD", "PCH"
 3 trials 
 1 / 1 / 0 / 0 💬 
 4 drugs 
 [ 3 drugs
 4 genes 
 45 pathways 
(1) About 100 patients
(2) 24 patients
Age distribution💬
 88 Chronic thromboembolic pulmonary hypertension [Resp] 💬
"CTEPH", "Idiopathic chronic pulmonary thromboembolism"
 157 trials 
 77 / 51 / 70 / 18 💬 
 107 drugs 
 [ 22 drugs
 14 genes 
 54 pathways 
(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 trials 
 14 / 18 / 8 / 2 💬 
 38 drugs 
 [ 15 drugs
 18 genes 
 118 pathways 
(1) 689 patients
(2) 912 patients
Age distribution💬
 228 Bronchiolitis obliterans [Resp] 💬
"Obliterating bronchiolitis"
 97 trials 
 49 / 40 / 36 / 16 💬 
 118 drugs 
 [ 32 drugs
 33 genes 
 156 pathways 
(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 trials 
 31 / 21 / 27 / 5 💬 
 22 drugs 
 [ 7 drugs
 3 genes 
 14 pathways 
(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 trials 
 0 / 2 / 2 / 0 💬 
 7 drugs 
 [ 4 drugs
 16 genes 
 26 pathways 
(1) About 3,000 patients
(2) 145 patients
Age distribution💬
 231 Alpha-1-antitrypsin deficiency [Resp] 💬
"AATD"
 93 trials 
 47 / 59 / 29 / 8 💬 
 77 drugs 
 [ 8 drugs
 10 genes 
 36 pathways 
(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 trials 
 2 / 2 / 1 / 0 💬 
 2 drugs 
 [ 2 drugs
 1 gene 
 51 pathways 
(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 trials 
 12 / 10 / 4 / 4 💬 
 22 drugs 
 [ 7 drugs
 5 genes 
 63 pathways 
(1) About 600 patients
(2) 17 patients
Age distribution💬
 294 Congenital diaphragmatic hernia [Resp] 💬
 16 trials 
 6 / 4 / 6 / 3 💬 
 26 drugs 
 [ 5 drugs
 5 genes 
 14 pathways 
(1) About 5,000 patients
(2) 13 patients
Age distribution💬
 330 Congenital tracheal stenosis [Resp] 💬
"Congenital subglottic stenosis"
 1 trial 
 0 / 1 / 0 / 0 💬 
 3 drugs 
 [ 2 drugs
 (1) About 1,000 patients
(2) 41 patients
Age distribution💬