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)
181	Crouzon syndrome [Hear] 💬	-	-	-	20 patients Age distribution💬
182	Apert syndrome [Hear] 💬	-	-	-	10 patients Age distribution💬
183	Pfeiffer syndrome [Hear] 💬 "Pfeiffer syndrome type 1", "Pfeiffer syndrome type 2", "Pfeiffer syndrome type 3"	-	-	-	5 patients Age distribution💬
184	Antley-Bixler syndrome [Hear] 💬	-	-	-	3 patients Age distribution💬
190	Branchio-oto-renal syndrome [Hear] 💬 "BOR syndrome"	-	-	-	7 patients Age distribution💬
303	Usher syndrome [Hear] 💬 [Eye] 💬	10 trials    | 6 / 10 / 2 / 0 💬	11 drugs   [ - ]	-	26 patients Age distribution💬
304	Juvenile-onset bilateral sensorineural hearing loss [Hear] 💬	-	-	-	53 patients Age distribution💬
305	Delayed endolymphatic hydrops [Hear] 💬	1 trial    | 0 / 0 / 0 / 0 💬	1 drug   [ 1 drug ]	-	28 patients Age distribution💬
306	Eosinophilic sinusitis [Imm] 💬 [Hear] 💬	-	-	-	22,340 patients Age distribution💬
330	Congenital tracheal stenosis [Resp] 💬 [Hear] 💬 "Congenital subglottic stenosis"	1 trial    | 0 / 1 / 0 / 0 💬	3 drugs   [ 2 drugs ]	-	44 patients Age distribution💬