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)
68	Ossification of the ligamentum flavum [Bone] 💬 "Ossification of ligamentum flavum", "OLF"	2 trials    | 0 / 0 / 0 / 0 💬	4 drugs   [ 4 drugs ]	1 gene   3 pathways	6,092 patients Age distribution💬
69	Ossification of posterior longitudinal ligament [Bone] 💬	2 trials    | 0 / 0 / 0 / 0 💬	2 drugs   [ 3 drugs ]	-	31,571 patients Age distribution💬
70	Spinal stenosis [Bone] 💬 "Extensive spinal canal stenosis"	95 trials    | 14 / 13 / 8 / 29 💬	169 drugs   [ 61 drugs ]	68 genes   90 pathways	4,874 patients Age distribution💬
71	Idiopathic osteonecrosis of the femoral head [Bone] 💬 "Idiopathic femoral head necrosis"	2 trials    | 0 / 1 / 0 / 0 💬	3 drugs   [ 3 drugs ]	3 genes   7 pathways	19,256 patients Age distribution💬
172	Hypophosphatasia [Bone] 💬	34 trials    | 13 / 20 / 4 / 5 💬	17 drugs   [ 4 drugs ]	3 genes   6 pathways	40 patients Age distribution💬
270	Chronic recurrent multifocal osteomyelitis [Bone] 💬	1 trial    | 0 / 0 / 0 / 0 💬	10 drugs   [ 9 drugs ]	6 genes   73 pathways	115 patients Age distribution💬
271	Ankylosing spondylitis [Bone] 💬 "Spondylarthritis ankylopoietica"	574 trials    | 236 / 145 / 234 / 113 💬	359 drugs   [ 68 drugs ]	41 genes   146 pathways	4,981 patients Age distribution💬
272	Fibrodysplasia ossificans progressiva [Bone] 💬 "FOP"	39 trials    | 17 / 26 / 15 / 3 💬	38 drugs   [ 7 drugs ]	27 genes   95 pathways	24 patients Age distribution💬
273	Congenital scoliosis with rib anomaly [Bone] 💬 "Congenital scoliosis"	-	-	-	21 patients Age distribution💬
274	Osteogenesis Imperfecta [Bone] 💬	91 trials    | 24 / 23 / 32 / 11 💬	101 drugs   [ 20 drugs ]	11 genes   48 pathways	152 patients Age distribution💬
275	Thanatophoric dysplasia [Bone] 💬	-	-	-	5 patients Age distribution💬
276	Achondroplasia [Bone] 💬	51 trials    | 27 / 39 / 13 / 6 💬	34 drugs   [ 6 drugs ]	4 genes   26 pathways	104 patients Age distribution💬