Disease The intractable diseases designated by MHLW, Japan

Diseases : 348 - Clinical trials : 39,610 / Drugs : 18,765 - ( DrugBank : 2,435 ) / Drug target genes : 703 - Drug target pathways : 305

ID	Disease name [Group]	Clinical trial Phase 1 / 2 / 3 / 4	Drug [ DrugBank ]	Target gene Target pathway	Domestic patients Med expenses recipients (FY2023)
66	IgA nephropathy [Kid] 💬 "IgA nephritis", "Berger disease", "IgA-IgG nephropathy" 66. IgA nephropathy × [Kid] Kidney and urinary diseases	349 trials    | 15 / 116 / 113 / 37 💬 66. IgA nephropathy × Some trials have no Phase data.	251 drugs   [ 75 drugs ]	43 genes   152 pathways	14,333 patients Age distribution💬 66. IgA nephropathy × Age Num 0-9 - 10-19 96 20-29 1,324 30-39 1,880 40-49 3,142 50-59 3,506 60-69 2,390 70-74 1,056 75- 939
67	Polycystic kidney disease [Kid] 💬 "PKD", "PCKD", "Polycystic kidney", "Autosomal dominant PKD", "ADPKD", "Autosomal recessive PKD", "ARPKD" 67. Polycystic kidney disease × [Kid] Kidney and urinary diseases	233 trials    | 16 / 74 / 97 / 15 💬 67. Polycystic kidney disease × Some trials have no Phase data.	188 drugs   [ 54 drugs ]	60 genes   165 pathways	13,427 patients Age distribution💬 67. Polycystic kidney disease × Age Num 0-9 - 10-19 3 20-29 194 30-39 1,099 40-49 3,085 50-59 4,197 60-69 2,733 70-74 1,054 75- 1,062
109	Atypical hemolytic uremic syndrome [Kid] 💬 "Atypical HUS", "aHUS" 109. Atypical hemolytic uremic syndrome × [Kid] Kidney and urinary diseases	123 trials    | 0 / 32 / 46 / 4 💬 109. Atypical hemolytic uremic syndrome × Some trials have no Phase data.	29 drugs   [ 6 drugs ]	2 genes   11 pathways	100 patients Age distribution💬 109. Atypical hemolytic uremic syndrome × Age Num 0-9 - 10-19 1 20-29 13 30-39 22 40-49 16 50-59 21 60-69 10 70-74 1 75- 16
218	Alport syndrome [Kid] 💬 218. Alport syndrome × [Kid] Kidney and urinary diseases	41 trials    | 3 / 19 / 9 / 1 💬 218. Alport syndrome × Some trials have no Phase data.	32 drugs   [ 19 drugs ]	8 genes   44 pathways	300 patients Age distribution💬 218. Alport syndrome × Age Num 0-9 - 10-19 2 20-29 77 30-39 48 40-49 61 50-59 53 60-69 47 70-74 7 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 2 20-29 - 30-39 - 40-49 - 50-59 - 60-69 - 70-74 - 75- -
220	Rapidly progressive glomerulonephritis [Kid] 💬 220. Rapidly progressive glomerulonephritis × [Kid] Kidney and urinary diseases	4 trials    | 0 / 1 / 2 / 0 💬 220. Rapidly progressive glomerulonephritis × Some trials have no Phase data.	9 drugs   [ 5 drugs ]	3 genes   14 pathways	1,370 patients Age distribution💬 220. Rapidly progressive glomerulonephritis × Age Num 0-9 - 10-19 - 20-29 24 30-39 23 40-49 40 50-59 117 60-69 199 70-74 257 75- 710
221	Anti-glomerular basement membrane disease [Kid] 💬 "Goodpasture syndrome" 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.	8 drugs   [ 4 drugs ]	2 genes   20 pathways	421 patients Age distribution💬 221. Anti-glomerular basement membrane disease × Age Num 0-9 - 10-19 1 20-29 9 30-39 5 40-49 12 50-59 55 60-69 78 70-74 74 75- 187
222	Primary nephrotic syndrome [Kid] 💬 "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN" 222. Primary nephrotic syndrome × [Kid] Kidney and urinary diseases	382 trials    | 32 / 132 / 80 / 36 💬 222. Primary nephrotic syndrome × Some trials have no Phase data.	278 drugs   [ 98 drugs ]	73 genes   197 pathways	13,694 patients Age distribution💬 222. Primary nephrotic syndrome × Age Num 0-9 7 10-19 83 20-29 1,831 30-39 1,523 40-49 1,837 50-59 2,187 60-69 2,106 70-74 1,456 75- 2,664
223	Primary membranoproliferative glomerulonephritis [Kid] 💬 "Primary MPGN", "Primary membranoproliferative glomerulonephritis type I", "Primary MPGN I", "Primary membranoproliferative glomerulonephritis type III", "Primary MPGN III" 223. Primary membranoproliferative glomerulonephritis × [Kid] Kidney and urinary diseases	-	-	-	398 patients Age distribution💬 223. Primary membranoproliferative glomerulonephritis × Age Num 0-9 - 10-19 5 20-29 62 30-39 39 40-49 45 50-59 69 60-69 58 70-74 49 75- 71
224	Purpura nephritis [Kid] 💬 "Henoch-Schönlein purpura nephritis", "HSPN" 224. Purpura nephritis × [Kid] Kidney and urinary diseases	16 trials    | 1 / 3 / 0 / 4 💬 224. Purpura nephritis × Some trials have no Phase data.	32 drugs   [ 21 drugs ]	15 genes   56 pathways	1,130 patients Age distribution💬 224. Purpura nephritis × Age Num 0-9 - 10-19 11 20-29 148 30-39 114 40-49 189 50-59 188 60-69 180 70-74 116 75- 184
225	Congenital nephrogenic diabetes insipidus [Kid] 💬 "Hereditary nephrogenic diabetes insipidus", "Nephrogenic diabetes insipidus" 225. Congenital nephrogenic diabetes insipidus × [Kid] Kidney and urinary diseases	17 trials    | 2 / 5 / 0 / 2 💬 225. Congenital nephrogenic diabetes insipidus × Some trials have no Phase data.	40 drugs   [ 19 drugs ]	33 genes   67 pathways	46 patients Age distribution💬 225. Congenital nephrogenic diabetes insipidus × Age Num 0-9 - 10-19 - 20-29 23 30-39 7 40-49 8 50-59 3 60-69 3 70-74 - 75- 2
226	Interstitial cystitis with Hunners ulcer [Kid] 💬 "Interstitial cystitis" 226. Interstitial cystitis with Hunners ulcer × [Kid] Kidney and urinary diseases	163 trials    | 12 / 63 / 26 / 9 💬 226. Interstitial cystitis with Hunners ulcer × Some trials have no Phase data.	158 drugs   [ 58 drugs ]	79 genes   142 pathways	1,116 patients Age distribution💬 226. Interstitial cystitis with Hunners ulcer × Age Num 0-9 - 10-19 - 20-29 13 30-39 26 40-49 93 50-59 165 60-69 263 70-74 196 75- 360
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 2 50-59 3 60-69 2 70-74 - 75- -
335	Nephronophthisis [Kid] 💬 "NPHP", "NPH" 335. Nephronophthisis × [Kid] Kidney and urinary diseases	1 trial    | 0 / 0 / 0 / 0 💬 335. Nephronophthisis × Some trials have no Phase data.	1 drug   [ - ]	-	22 patients Age distribution💬 335. Nephronophthisis × Age Num 0-9 - 10-19 1 20-29 18 30-39 2 40-49 1 50-59 - 60-69 - 70-74 - 75- -