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

ID	Disease name [Group]	Clinical trial Phase 1 / 2 / 3 / 4	Drug [ DrugBank ]	Target gene Target pathway	Domestic patients Med expenses recipients (FY2022)
66	IgA nephropathy [Kid] 💬 "IgA nephritis", "Berger disease", "IgA-IgG nephropathy"	275 trials    | 30 / 87 / 97 / 30 💬	258 drugs   [ 82 drugs ]	36 genes   140 pathways	13,354 patients Age distribution💬
67	Polycystic kidney disease [Kid] 💬 "PKD", "PCKD", "Polycystic kidney", "Autosomal dominant PKD", "ADPKD", "Autosomal recessive PKD", "ARPKD"	221 trials    | 97 / 78 / 110 / 29 💬	212 drugs   [ 55 drugs ]	40 genes   151 pathways	12,790 patients Age distribution💬
109	Atypical hemolytic uremic syndrome [Kid] 💬 "aHUS"	114 trials    | 75 / 41 / 54 / 18 💬	36 drugs   [ 7 drugs ]	3 genes   11 pathways	81 patients Age distribution💬
218	Alport syndrome [Kid] 💬 "X chromosome-linked Alport syndrome", "Autosomal recessive Alport syndrome"	30 trials    | 11 / 20 / 9 / 3 💬	36 drugs   [ 15 drugs ]	8 genes   46 pathways	258 patients Age distribution💬
219	Galloway-Mowat syndrome [Kid] 💬	-	-	-	1 patient Age distribution💬
220	Rapidly progressive glomerulonephritis [Kid] 💬	2 trials    | 0 / 1 / 2 / 0 💬	4 drugs   [ 2 drugs ]	1 gene   1 pathway	1,245 patients Age distribution💬
221	Anti-glomerular basement membrane disease [Kid] 💬	1 trial    | 0 / 1 / 0 / 0 💬	3 drugs   [ 1 drug ]	-	364 patients Age distribution💬
222	Primary nephrotic syndrome [Kid] 💬 "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN"	310 trials    | 96 / 128 / 65 / 43 💬	295 drugs   [ 117 drugs ]	63 genes   194 pathways	12,889 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"	-	-	-	373 patients Age distribution💬
224	Purpura nephritis [Kid] 💬	16 trials    | 2 / 3 / 0 / 4 💬	43 drugs   [ 21 drugs ]	15 genes   58 pathways	1,037 patients Age distribution💬
225	Congenital nephrogenic diabetes insipidus [Kid] 💬 "Hereditary nephrogenic diabetes insipidus", "Nephrogenic diabetes insipidus"	15 trials    | 7 / 5 / 1 / 3 💬	48 drugs   [ 18 drugs ]	31 genes   65 pathways	53 patients Age distribution💬
226	Interstitial cystitis with Hunners ulcer [Kid] 💬 "Interstitial cystitis"	145 trials    | 48 / 58 / 27 / 17 💬	156 drugs   [ 51 drugs ]	64 genes   146 pathways	1,016 patients Age distribution💬
315	Nail-Patella syndrome [Kid] 💬 "LMX1B-associated nephropathy"	-	-	-	12 patients Age distribution💬
335	Nephronophthisis [Kid] 💬 "NPHP", "NPH"	1 trial    | 0 / 0 / 0 / 0 💬	1 drug   [ - ]	-	17 patients Age distribution💬