Disease The intractable diseases designated by MHLW, Japan

Diseases : 348 - Clinical trials : 43,690 / Drugs : 27,527 - ( DrugBank : 2,441 ) / Drug target genes : 714 - Drug target pathways : 315

ID	Disease name [Group]	Clinical trial Phase 1 / 2 / 3 / 4	Drug [ DrugBank ]	Target gene Target pathway	Domestic patients Med expenses recipients, FY2024 (corrected)
66	IgA nephropathy [Kid] 💬 "IgA nephritis", "Berger disease", "IgA-IgG nephropathy", "Glomerulonephritis" 66. IgA nephropathy × [Kid] Kidney and urinary diseases	407 trials    | 21 / 129 / 123 / 43 💬 66. IgA nephropathy × Some trials have no Phase data.	342 drugs   [ 73 drugs ]	40 genes   146 pathways	15,355 patients Age distribution💬 66. IgA nephropathy × Age Num 0-9 - 10-19 101 20-29 1,251 30-39 2,078 40-49 3,261 50-59 3,802 60-69 2,608 70-74 1,067 75- 1,187
67	Polycystic kidney disease [Kid] 💬 "PKD", "PCKD", "Polycystic kidney", "Autosomal dominant polycystic kidney disease", "Autosomal dominant PKD", "ADPKD", "Autosomal recessive polycystic kidney disease", "Autosomal recessive PKD", "ARPKD" 67. Polycystic kidney disease × [Kid] Kidney and urinary diseases	251 trials    | 23 / 78 / 100 / 15 💬 67. Polycystic kidney disease × Some trials have no Phase data.	278 drugs   [ 69 drugs ]	97 genes   182 pathways	13,912 patients Age distribution💬 67. Polycystic kidney disease × Age Num 0-9 - 10-19 5 20-29 187 30-39 1,088 40-49 3,118 50-59 4,379 60-69 2,895 70-74 1,059 75- 1,181
109	Atypical hemolytic uremic syndrome [Kid] 💬 "Atypical HUS", "aHUS" 109. Atypical hemolytic uremic syndrome × [Kid] Kidney and urinary diseases	130 trials    | 0 / 32 / 49 / 6 💬 109. Atypical hemolytic uremic syndrome × Some trials have no Phase data.	44 drugs   [ 5 drugs ]	2 genes   11 pathways	112 patients Age distribution💬 109. Atypical hemolytic uremic syndrome × Age Num 0-9 - 10-19 2 20-29 15 30-39 21 40-49 22 50-59 20 60-69 14 70-74 7 75- 11
218	Alport syndrome [Kid] 💬 "Glomerulonephritis" 218. Alport syndrome × [Kid] Kidney and urinary diseases	47 trials    | 3 / 21 / 9 / 3 💬 218. Alport syndrome × Some trials have no Phase data.	49 drugs   [ 23 drugs ]	8 genes   46 pathways	326 patients Age distribution💬 218. Alport syndrome × Age Num 0-9 - 10-19 - 20-29 86 30-39 46 40-49 65 50-59 67 60-69 48 70-74 9 75- 5
219	Galloway-Mowat syndrome [Kid] 💬 219. Galloway-Mowat syndrome × [Kid] Kidney and urinary diseases	-	-	-	2 patients Age distribution💬 219. Galloway-Mowat syndrome × Age Num 0-9 - 10-19 - 20-29 2 30-39 - 40-49 - 50-59 - 60-69 - 70-74 - 75- -
220	Rapidly progressive glomerulonephritis [Kid] 💬 "RPGN", "Necrotizing crescentic glomerulonephritis", "NCGN", "Glomerulonephritis" 220. Rapidly progressive glomerulonephritis × [Kid] Kidney and urinary diseases	-	-	-	1,440 patients Age distribution💬 220. Rapidly progressive glomerulonephritis × Age Num 0-9 - 10-19 1 20-29 24 30-39 18 40-49 39 50-59 121 60-69 216 70-74 250 75- 771
221	Anti-glomerular basement membrane disease [Kid] 💬 "Goodpasture syndrome", "Glomerulonephritis" 221. Anti-glomerular basement membrane disease × [Kid] Kidney and urinary diseases	9 trials    | 0 / 3 / 1 / 0 💬 221. Anti-glomerular basement membrane disease × Some trials have no Phase data.	12 drugs   [ 4 drugs ]	2 genes   22 pathways	458 patients Age distribution💬 221. Anti-glomerular basement membrane disease × Age Num 0-9 - 10-19 1 20-29 10 30-39 11 40-49 16 50-59 58 60-69 83 70-74 78 75- 201
222	Primary nephrotic syndrome [Kid] 💬 "Nephrotic syndrome", "Glomerulonephritis", "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Crescentic glomerulonephritis", "CGN", "CrGN", "Endocapillary proliferative glomerulonephritis" 222. Primary nephrotic syndrome × [Kid] Kidney and urinary diseases	576 trials    | 44 / 151 / 117 / 86 💬 222. Primary nephrotic syndrome × Some trials have no Phase data.	519 drugs   [ 130 drugs ]	108 genes   219 pathways	14,333 patients Age distribution💬 222. Primary nephrotic syndrome × Age Num 0-9 2 10-19 70 20-29 1,819 30-39 1,561 40-49 1,848 50-59 2,318 60-69 2,227 70-74 1,492 75- 2,996
223	Primary membranoproliferative glomerulonephritis [Kid] 💬 "Primary MPGN", "Primary membranoproliferative glomerulonephritis type I", "Primary MPGN I", "Primary membranoproliferative glomerulonephritis type III", "Primary MPGN III", "Membranoproliferative glomerulonephritis", "Glomerulonephritis", "C3 glomerulonephritis", "C3 glomerulopathy", "IC-MPGN", "MPGN" 223. Primary membranoproliferative glomerulonephritis × [Kid] Kidney and urinary diseases	243 trials    | 8 / 62 / 49 / 17 💬 223. Primary membranoproliferative glomerulonephritis × Some trials have no Phase data.	296 drugs   [ 84 drugs ]	46 genes   169 pathways	440 patients Age distribution💬 223. Primary membranoproliferative glomerulonephritis × Age Num 0-9 - 10-19 3 20-29 74 30-39 42 40-49 44 50-59 77 60-69 72 70-74 45 75- 83
224	Purpura nephritis [Kid] 💬 "Henoch-Schönlein purpura nephritis", "HSPN" 224. Purpura nephritis × [Kid] Kidney and urinary diseases	19 trials    | 1 / 3 / 1 / 5 💬 224. Purpura nephritis × Some trials have no Phase data.	42 drugs   [ 19 drugs ]	16 genes   64 pathways	1,237 patients Age distribution💬 224. Purpura nephritis × Age Num 0-9 - 10-19 9 20-29 160 30-39 128 40-49 214 50-59 200 60-69 190 70-74 105 75- 231
225	Congenital nephrogenic diabetes insipidus [Kid] 💬 "Hereditary nephrogenic diabetes insipidus", "Nephrogenic diabetes insipidus" 225. Congenital nephrogenic diabetes insipidus × [Kid] Kidney and urinary diseases	19 trials    | 3 / 5 / 0 / 2 💬 225. Congenital nephrogenic diabetes insipidus × Some trials have no Phase data.	56 drugs   [ 21 drugs ]	33 genes   67 pathways	59 patients Age distribution💬 225. Congenital nephrogenic diabetes insipidus × Age Num 0-9 - 10-19 2 20-29 27 30-39 10 40-49 8 50-59 5 60-69 4 70-74 1 75- 2
226	Interstitial cystitis with Hunners ulcer [Kid] 💬 "Interstitial cystitis" 226. Interstitial cystitis with Hunners ulcer × [Kid] Kidney and urinary diseases	177 trials    | 15 / 67 / 28 / 9 💬 226. Interstitial cystitis with Hunners ulcer × Some trials have no Phase data.	208 drugs   [ 60 drugs ]	87 genes   148 pathways	1,161 patients Age distribution💬 226. Interstitial cystitis with Hunners ulcer × Age Num 0-9 - 10-19 - 20-29 12 30-39 24 40-49 80 50-59 169 60-69 279 70-74 187 75- 410
315	Nail-Patella syndrome [Kid] 💬 "LMX1B-associated nephropathy" 315. Nail-Patella syndrome × [Kid] Kidney and urinary diseases	-	-	-	10 patients Age distribution💬 315. Nail-Patella syndrome × Age Num 0-9 - 10-19 - 20-29 2 30-39 1 40-49 1 50-59 3 60-69 3 70-74 - 75- -
335	Nephronophthisis [Kid] 💬 "NPHP", "NPH" 335. Nephronophthisis × [Kid] Kidney and urinary diseases	2 trials    | 0 / 0 / 0 / 0 💬 335. Nephronophthisis × Some trials have no Phase data.	3 drugs   [ - ]	-	27 patients Age distribution💬 335. Nephronophthisis × Age Num 0-9 - 10-19 1 20-29 23 30-39 2 40-49 1 50-59 - 60-69 - 70-74 - 75- -