Disease - Clinical trilas for the 333 intractable diseases designated by the Ministry of Health, Labour and Welfare (MHLW), Japan💬 Info on mouse-over

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ID	Disease name	Clinical trial Phase 1 / 2 / 3 / 4	Drug [ DrugBank ]	Target gene Target pathway	Domestic patients (1) MHLW, (2) Med expenses recipients (FY2020)	Related info (in Japanese)
1	Spinal and bulbar muscular atrophy "Spinobulbar muscular atrophy", "SBMA", "Kennedy disease", "Kennedy-Alter-Sung syndrome"	17 trials   0 / 9 / 2 / 1 💬	16 drugs   [ 8 drugs ]	10 genes   15 pathways	(1) 1,223 patients (2) 1,622 patients Age distribution💬	Biobank Animal model
2	Amyotrophic lateral sclerosis "ALS"	508 trials   252 / 235 / 151 / 35 💬	530 drugs   [ 146 drugs ]	170 genes   221 pathways	(1) 9,096 patients (2) 10,514 patients Age distribution💬	Biobank Animal model
3	Spinal muscular atrophy "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"	179 trials   141 / 107 / 75 / 28 💬	102 drugs   [ 26 drugs ]	52 genes   78 pathways	(1) 712 patients (2) 921 patients Age distribution💬	Biobank Pediatric diseases
4	Primary lateral sclerosis "PLS"	6 trials   3 / 2 / 0 / 1 💬	16 drugs   [ 8 drugs ]	19 genes   32 pathways	(1) 175 patients (By the research group) (2) 127 patients Age distribution💬	Biobank
5	Progressive supranuclear palsy "PSP"	82 trials   48 / 35 / 12 / 6 💬	107 drugs   [ 36 drugs ]	60 genes   90 pathways	(1) About 8,100 patients (2) 12,766 patients Age distribution💬	Biobank
6	Parkinson disease	2,123 trials   864 / 632 / 514 / 272 💬	2,046 drugs   [ 324 drugs ]	183 genes   198 pathways	(1) About 108,800 patients (estimated from Parkinson disease related diseases) (2) 142,375 patients Age distribution💬	Biobank
7	Corticobasal degeneration "Corticobasal syndrome", "CBD"	16 trials   5 / 2 / 1 / 0 💬	33 drugs   [ 11 drugs ]	6 genes   13 pathways	(1) 3,500 patients (2) 4,749 patients Age distribution💬
8	Huntington disease "Huntington chorea"	197 trials   127 / 103 / 42 / 20 💬	171 drugs   [ 54 drugs ]	82 genes   144 pathways	(1) 933 patients (2) 950 patients Age distribution💬	Biobank Animal model
9	Neuroacanthocytosis "Choreoacanthocytosis", "Chorea-acanthocytosis", "Levine-Critchley syndrome", "McLeod syndrome", "Huntington disease-like 2", "HDL2", "Pantothenate kinase-associated neurodegeneration", "PKAN", "Hallervorden-Spatz syndrome"  collapse"Choreoacanthocytosis", "Chorea-acanthocytosis", "Levine-Critchley syndrome", "McLeod syndrome", "Huntington disease-like 2", "HDL2", "Pantothenate ki ... show all	-	-	-	(1) Less than 100 patients (By the research group) (2) 33 patients Age distribution💬	Pediatric diseases
10	Charcot-Marie-Tooth disease "CMT"	31 trials   23 / 16 / 16 / 4 💬	38 drugs   [ 9 drugs ]	11 genes   14 pathways	(1) 6,250 patients (By the research group) (2) 774 patients Age distribution💬	Biobank Animal model Pediatric diseases

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