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

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Showing 1 to 10 of 338 diseases

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ID	Disease name [Group]	Clinical trial Phase 1 / 2 / 3 / 4	Drug [ DrugBank ]	Target gene Target pathway	Domestic patients Med expenses recipients (FY2022)
1	Spinal and bulbar muscular atrophy [Neu] 💬 "Spinobulbar muscular atrophy", "SBMA", "Kennedy disease", "Kennedy-Alter-Sung syndrome"	18 trials    | 1 / 10 / 2 / 1 💬	15 drugs   [ 8 drugs ]	10 genes   17 pathways	1,697 patients Age distribution💬   1. Spinal and bulbar muscular atrophy × Age Num 0-9 - 10-19 - 20-29 1 30-39 33 40-49 176 50-59 409 60-69 526 70-74 269 75- 283
2	Amyotrophic lateral sclerosis [Neu] 💬 "ALS"	645 trials    | 227 / 290 / 231 / 26 💬	589 drugs   [ 163 drugs ]	150 genes   225 pathways	9,765 patients Age distribution💬   2. Amyotrophic lateral sclerosis × Age Num 0-9 - 10-19 1 20-29 14 30-39 111 40-49 506 50-59 1,316 60-69 2,359 70-74 2,018 75- 3,440
3	Spinal muscular atrophy [Neu] 💬 "Myelopathic muscular atrophy", "Spinal muscular atrophy type I", "SMA I", "Werdnig-Hoffman disease", "Spinal muscular atrophy type II", "SMA II", "Dubowitz disease", "Spinal muscular atrophy type III", "SMA III", "Kugelberg-Welander disease", "Spinal muscular atrophy type IV", "SMA IV"  "Myelopathic muscular atrophy", "Spinal muscular atrophy type I", "SMA I", "Werdnig-Hoffman disease" ...	237 trials    | 106 / 117 / 116 / 28 💬	123 drugs   [ 29 drugs ]	51 genes   75 pathways	955 patients Age distribution💬   3. Spinal muscular atrophy × Age Num 0-9 8 10-19 56 20-29 151 30-39 89 40-49 114 50-59 131 60-69 169 70-74 90 75- 147
4	Primary lateral sclerosis [Neu] 💬 "PLS"	5 trials    | 2 / 2 / 0 / 1 💬	13 drugs   [ 6 drugs ]	13 genes   25 pathways	146 patients Age distribution💬   4. Primary lateral sclerosis × Age Num 0-9 - 10-19 - 20-29 - 30-39 - 40-49 8 50-59 16 60-69 31 70-74 34 75- 57
5	Progressive supranuclear palsy [Neu] 💬 "PSP"	89 trials    | 45 / 42 / 9 / 6 💬	107 drugs   [ 40 drugs ]	65 genes   108 pathways	12,830 patients Age distribution💬   5. Progressive supranuclear palsy × Age Num 0-9 - 10-19 - 20-29 1 30-39 - 40-49 6 50-59 124 60-69 1,209 70-74 2,312 75- 9,178
6	Parkinson disease [Neu] 💬 "Disease Parkinson's"	2,307 trials    | 810 / 703 / 607 / 290 💬	2,007 drugs   [ 349 drugs ]	188 genes   199 pathways	143,267 patients Age distribution💬   6. Parkinson disease × Age Num 0-9 - 10-19 4 20-29 17 30-39 138 40-49 1,109 50-59 5,598 60-69 19,866 70-74 26,688 75- 89,847
7	Corticobasal degeneration [Neu] 💬 "Corticobasal syndrome", "CBD"	16 trials    | 5 / 3 / 1 / 0 💬	28 drugs   [ 13 drugs ]	9 genes   38 pathways	4,428 patients Age distribution💬   7. Corticobasal degeneration × Age Num 0-9 - 10-19 - 20-29 - 30-39 1 40-49 12 50-59 117 60-69 604 70-74 971 75- 2,723
8	Huntington disease [Neu] 💬 "Huntington chorea"	242 trials    | 111 / 134 / 50 / 13 💬	205 drugs   [ 62 drugs ]	85 genes   159 pathways	892 patients Age distribution💬   8. Huntington disease × Age Num 0-9 - 10-19 1 20-29 8 30-39 34 40-49 133 50-59 210 60-69 207 70-74 124 75- 175
9	Neuroacanthocytosis [Neu] 💬 "Choreoacanthocytosis", "Chorea-acanthocytosis", "Levine-Critchley syndrome", "McLeod syndrome", "Huntington disease-like 2", "HDL2", "Pantothenate kinase-associated neurodegeneration", "PKAN", "Hallervorden-Spatz syndrome"  "Choreoacanthocytosis", "Chorea-acanthocytosis", "Levine-Critchley syndrome", "McLeod syndrome", "Hu ...	-	-	-	36 patients Age distribution💬   9. Neuroacanthocytosis × Age Num 0-9 - 10-19 - 20-29 1 30-39 8 40-49 8 50-59 9 60-69 6 70-74 4 75- -
10	Charcot-Marie-Tooth disease [Neu] 💬 "CMT", "Charcot-Marie-Tooth disease type 1", "CMT1", "Demyelinating CMT", "Charcot-Marie-Tooth disease type 2", "CMT2", "Axonal CMT", "Intermediate Charcot-Marie-Tooth disease", "CMT-I", "Intermediate CMT"  "CMT", "Charcot-Marie-Tooth disease type 1", "CMT1", "Demyelinating CMT", "Charcot-Marie-Tooth disea ...	41 trials    | 25 / 17 / 24 / 4 💬	46 drugs   [ 9 drugs ]	11 genes   15 pathways	845 patients Age distribution💬   10. Charcot-Marie-Tooth disease × Age Num 0-9 2 10-19 12 20-29 37 30-39 40 40-49 105 50-59 181 60-69 188 70-74 114 75- 166

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