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: Kidney and urinary 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) |
|---|---|---|---|---|---|
| 66 | IgA nephropathy [Kid] 💬 "IgA nephritis", "Berger disease", "IgA-IgG nephropathy" | 255 255 trials 33 / 81 / 93 / 30 💬 | 255 255 drugs [ 79 79 drugs ] | 35 35 genes 137 pathways | 12447 (1) About 33,000 patients (By the research group) (2) 12,447 patients Age distribution  💬 |
| 67 | Polycystic kidney disease [Kid] 💬 "PKD", "PCKD", "Polycystic kidney", "Autosomal dominant PKD", "ADPKD", "Autosomal recessive PKD", "ARPKD" | 216 216 trials 94 / 77 / 108 / 27 💬 | 219 219 drugs [ 50 50 drugs ] | 39 39 genes 151 pathways | 12164 (1) About 31,000 patients (By the research group) (2) 12,164 patients Age distribution  💬 |
| 109 | Atypical hemolytic uremic syndrome [Kid] 💬 "aHUS" | 115 115 trials 80 / 40 / 54 / 19 💬 | 37 37 drugs [ 7 7 drugs ] | 3 3 genes 10 pathways | 78 (1) Less than 200 patients (By the research group) (2) 78 patients Age distribution  💬 |
| 218 | Alport syndrome [Kid] 💬 "X chromosome-linked Alport syndrome", "Autosomal recessive Alport syndrome" | 26 26 trials 11 / 17 / 8 / 2 💬 | 33 33 drugs [ 13 13 drugs ] | 6 6 genes 29 pathways | 223 (1) About 1,200 patients (2) 223 patients Age distribution  💬 |
| 219 | Galloway-Mowat syndrome [Kid] 💬 | 0 - | 0 - | 0 - | 1 (1) About 200 patients (2) 1 patient Age distribution  💬 |
| 220 | Rapidly progressive glomerulonephritis [Kid] 💬 | 2 2 trials 0 / 1 / 2 / 0 💬 | 5 5 drugs [ 2 2 drugs ] | 1 1 gene 1 pathway | 1147 (1) About 3,800~5,800 patients in total estimated (2) 1,147 patients Age distribution  💬 |
| 221 | Anti-glomerular basement membrane disease [Kid] 💬 | 1 1 trial 0 / 1 / 0 / 0 💬 | 3 3 drugs [ 1 1 drug ] | 0 - | 343 (1) About 200~400 patients (2) 343 patients Age distribution  💬 |
| 222 | Primary nephrotic syndrome [Kid] 💬 "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN" | 285 285 trials 93 / 128 / 63 / 39 💬 | 285 285 drugs [ 108 108 drugs ] | 62 62 genes 191 pathways | 12221 (1) About 16,000 patients (2) 12,221 patients Age distribution  💬 |
| 223 | Primary membranoproliferative glomerulonephritis [Kid] 💬 "Primary MPGN", "Primary membranoproliferative glomerulonephritis type I", "Primary MPGN I", "Primary membranoproliferative glomerulonephritis type III", "Primary MPGN III", "Mesangial proliferative primary membranoproliferative glomerulonephritis type I", "Mesangial proliferative primary MPGN I", "Primary membranoproliferative glomerulonephritis type I, chronic / nest-like type", "Primary MPGN I, chronic / nest-like type", "Primary membranoproliferative glomerulonephritis type I, acute / nest-like type", "Primary MPGN I, acute / nest-like type", "Primary membranoproliferative glomerulonephritis type I, chronic / diffuse type", "Primary MPGN I, chronic / diffuse type", "Primary membranoproliferative glomerulonephritis type I, non-lobed type", "Primary MPGN I, non-lobed type", "Primary membranoproliferative glomerulonephritis type I, acute / diffuse type", "Primary MPGN I, acute / diffuse type", "Primary membranoproliferative glomerulonephritis type I, intraductal proliferation type", "Primary MPGN I, intraductal proliferation type", "Primary membranoproliferative glomerulonephritis type I, lobation type", "Primary MPGN I, lobation type", "Primary membranoproliferative glomerulonephritis type I, end-of-life type", "Primary MPGN I, end-of-life type" | 0 - | 0 - | 0 - | 367 (1) About 1,000 patients (2) 367 patients Age distribution  💬 |
| 224 | Purpura nephritis [Kid] 💬 | 13 13 trials 2 / 3 / 0 / 2 💬 | 42 42 drugs [ 20 20 drugs ] | 15 15 genes 57 pathways | 1023 (1) 400~640 cases / year (2) 1,023 patients Age distribution  💬 |
| 225 | Congenital nephrogenic diabetes insipidus [Kid] 💬 "Hereditary nephrogenic diabetes insipidus", "Nephrogenic diabetes insipidus" | 15 15 trials 7 / 5 / 1 / 3 💬 | 48 48 drugs [ 18 18 drugs ] | 31 31 genes 65 pathways | 45 (1) About 200 patients (2) 45 patients Age distribution  💬 |
| 226 | Interstitial cystitis with Hunners ulcer [Kid] 💬 "Interstitial cystitis" | 143 143 trials 50 / 55 / 25 / 17 💬 | 171 171 drugs [ 56 56 drugs ] | 64 64 genes 145 pathways | 885 (1) About 2,000 patients (2) 885 patients Age distribution  💬 |
| 315 | Nail-Patella syndrome [Kid] 💬 "LMX1B-associated nephropathy" | 0 - | 0 - | 0 - | 7 (1) About 500 patients (2) 7 patients Age distribution  💬 |
| 335 | Nephronophthisis [Kid] 💬 "NPHP", "NPH" | 0 - | 0 - | 0 - | 3 (1) About 130-220 patients (2) 3 patients Age distribution  💬 |