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: Cardiovascular 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) |
|---|---|---|---|---|---|
| 57 | Idiopathic dilated cardiomyopathy [Card] 💬 | 12 12 trials 3 / 6 / 1 / 3 💬 | 25 25 drugs [ 12 12 drugs ] | 9 9 genes 34 pathways | 18724 (1) 27,968 patients (2) 18,724 patients Age distribution  💬 |
| 58 | Hypertrophic cardiomyopathy [Card] 💬 "HCM", "Nonobstructive hypertrophic cardiomyopathy", "Nonobstructive HCM", "Obstructive hypertrophic cardiomyopathy", "Obstructive HCM", "OHCM", "Hypertrophic obstructive cardiomyopathy", "HOCM", "Mid-ventricular obstructive hypertrophic cardiomyopathy", "MVOHCM", "Mid-ventricular hypertrophic obstructive cardiomyopathy", "MVHOCM", "Apical hypertrophic cardiomyopathy", "APH", "Expansion phase hypertrophic cardiomyopathy" | 119 119 trials 27 / 52 / 34 / 14 💬 | 163 163 drugs [ 45 45 drugs ] | 48 48 genes 161 pathways | 4201 (1) 4,667 patients (2) 4,201 patients Age distribution  💬 |
| 59 | Restricted cardiomyopathy [Card] 💬 "Restrictive cardiomyopathy", "Constrictive cardiomyopathy" | 0 - | 0 - | 0 - | 60 (1) 50 patients (2) 60 patients Age distribution  💬 |
| 207 | Persistent truncus arteriosus [Card] 💬 "Persistent truncus arteriosus type I", "Persistent truncus arteriosus type II", "Persistent truncus arteriosus type III", "Persistent truncus arteriosus type IV" | 0 - | 0 - | 0 - | 32 (1) About 500 patients (2) 32 patients Age distribution  💬 |
| 208 | Corrected transposition of great arteries [Card] 💬 | 0 - | 0 - | 0 - | 186 (1) [Disease ID 208-209 total] About 900 patients (2) 186 patients Age distribution  💬 |
| 209 | Complete transposition of great vessel [Card] 💬 "Complete transposition of great arteries", "Complete TGA", "Complete transposition of great vessel type I", "Complete transposition of great arteries type I", "Complete TGA type I", "Complete transposition of great vessel type II", "Complete transposition of great arteries type II", "Complete TGA type II", "Complete transposition of great vessel type III", "Complete transposition of great arteries type III", "Complete TGA type III", "Complete transposition of great vessel type IV", "Complete transposition of great arteries type IV", "Complete TGA type IV" | 0 - | 0 - | 0 - | 244 (1) [Disease ID 208-209 total] About 900 patients (2) 244 patients Age distribution  💬 |
| 210 | Single Ventricle [Card] 💬 "SV", "Single ventricle heart defect", "Univentricular heart", "UVH", "Single ventricular circulation syndrome" | 49 49 trials 22 / 14 / 23 / 6 💬 | 53 53 drugs [ 23 23 drugs ] | 32 32 genes 67 pathways | 425 (1) [Disease ID 210-213 total] About 3,500 patients (Patients of single ventricle heart defects) (2) 425 patients Age distribution  💬 |
| 211 | Hypoplastic left heart syndrome [Card] 💬 "HLHS" | 21 21 trials 11 / 10 / 0 / 0 💬 | 29 29 drugs [ 9 9 drugs ] | 5 5 genes 14 pathways | 54 (1) [Disease ID 210-213 total] About 3,500 patients (Patients of single ventricle heart defects) (2) 54 patients Age distribution  💬 |
| 212 | Tricuspid atresia [Card] 💬 "TA" | 6 6 trials 2 / 2 / 0 / 0 💬 | 8 8 drugs [ 7 7 drugs ] | 8 8 genes 12 pathways | 183 (1) [Disease ID 210-213 total] About 3,500 patients (Patients of single ventricle heart defects) (2) 183 patients Age distribution  💬 |
| 213 | Pulmonary atresia without ventricular septum defect [Card] 💬 "Pulmonary atresia with intact ventricular septum", "Pulmonary atresia" | 0 - | 0 - | 0 - | 140 (1) [Disease ID 210-213 total] About 3,500 patients (Patients of single ventricle heart defects) (2) 140 patients Age distribution  💬 |
| 214 | Pulmonary atresia with ventricular septum defect [Card] 💬 "PAVSD", "Pulmonary atresia with ventricular septal defect", "Pulmonary atresia", "Tetralogy of Fallot with pulmonary atresia", "TFPA" | 2 2 trials 0 / 0 / 0 / 0 💬 | 4 4 drugs [ 4 4 drugs ] | 0 - | 108 (1) [Disease ID 214-216 total] About 5,500 patients (Patients of tetralogy of Fallot related diseases) (2) 108 patients Age distribution  💬 |
| 215 | Tetralogy of Fallot [Card] 💬 "Fallot tetralogy" | 22 22 trials 5 / 4 / 1 / 1 💬 | 48 48 drugs [ 19 19 drugs ] | 18 18 genes 49 pathways | 689 (1) [Disease ID 214-216 total] About 5,500 patients (Patients of tetralogy of Fallot related diseases) (2) 689 patients Age distribution  💬 |
| 216 | Double outlet right ventricle [Card] 💬 | 0 - | 0 - | 0 - | 256 (1) [Disease ID 214-216 total] About 5,500 patients (Patients of tetralogy of Fallot related diseases) (2) 256 patients Age distribution  💬 |
| 217 | Ebstein disease [Card] 💬 "Ebstein malformation" | 0 - | 0 - | 0 - | 130 (1) About 300 patients (2) 130 patients Age distribution  💬 |
| 279 | Huge venous malformation with cervical, oral and pharyngeal diffuse lesion [Card] 💬 oral and pharyngeal diffuse lesion", "Huge venous malformation", "Venous malformation" | 14 14 trials 2 / 6 / 1 / 2 💬 | 24 24 drugs [ 9 9 drugs ] | 3 3 genes 105 pathways | 47 (1) About 200 patients (2) 47 patients Age distribution  💬 |
| 280 | Huge arteriovenous malformation with cervicofacial or limb lesion [Card] 💬 "Huge arteriovenous malformation", "Arteriovenous malformation" | 25 25 trials 14 / 7 / 2 / 6 💬 | 29 29 drugs [ 15 15 drugs ] | 14 14 genes 153 pathways | 104 (1) About 700 patients (2) 104 patients Age distribution  💬 |
| 281 | Klippel-Trenaunay-Weber syndrome [Card] 💬 "Klippel-Trenauney-Weber syndrome", "Klippel-Trenaunay syndrome", "Klippel-Trenauney syndrome", "KTS", "Parkes Weber syndrome", "PWS" | 1 1 trial 0 / 0 / 0 / 0 💬 | 1 1 drug [ 1 1 drug ] | 1 1 gene 51 pathways | 239 (1) About 3,000 patients (2) 239 patients Age distribution  💬 |
| 311 | Congenital tricuspid stenosis [Card] 💬 | 0 - | 0 - | 0 - | 3 (1) About 500 patients (2) 3 patients Age distribution  💬 |
| 312 | Congenital mitral stenosis [Card] 💬 | 0 - | 0 - | 0 - | 12 (1) About 100 patients (2) 12 patients Age distribution  💬 |
| 313 | Congenital pulmonary vein stenosis [Card] 💬 | 0 - | 0 - | 0 - | 1 (1) About 80 patients (2) 1 patient Age distribution  💬 |
| 314 | Vascular sling [Card] 💬 | 0 - | 0 - | 0 - | 2 (1) About 600 patients (2) 2 patients Age distribution  💬 |