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No. 薬物名（臨床試験情報から抽出） DrugBank KEGG DRUG KEGG GENES KEGG PATHWAY 指定難病告示番号
1(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one----1件: 240
25,6,7,8-tetrahydrobiopterin1件: Sapropterin1件: D08505 --1件: 240
36R-BH41件: Sapropterin1件: D08505 --3件: 86, 124, 240
4A16AX07----1件: 240
5Amino acids1件: Amino acids---3件: 70, 240, 298
6Behavioral: Neuro-psychological Assessment----1件: 240
7Behavioral: phenylalanine restricted diet1件: Phenylalanine1件: D00021 --1件: 240
8Behavioral: Restricted phenylalanine diet1件: Phenylalanine1件: D00021 --1件: 240
9BH4----5件: 51, 86, 124, 240, 299
10Blood samples----9件: 13, 41, 46, 51, 61, 97, 227, 240, 271
11BMN 165----1件: 240
12BMN 165 (rAvPAL-PEG)1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
13BMN 307----1件: 240
14BMN165 20mg/day----1件: 240
15BMN165 40mg/day----1件: 240
16CDX----2件: 222, 240
17CDX 6114----1件: 240
18CNSA-001----1件: 240
19Cohort 1 0.225g----1件: 240
20Cohort 2 0.75g----1件: 240
21Cohort 3 2.25 g----1件: 240
22Corn1件: Corn---2件: 46, 240
23Corn/soy oil1件: Corn---1件: 240
24Diagnostic Test: Whole body Phe oxidation testing----1件: 240
25Diet----3件: 97, 240, 299
26Dietary Supplement for PKU patients----1件: 240
27Docosahexaenoic acid1件: Doconexent---7件: 6, 49, 90, 94, 240, 299, 301
28Free amino acids intake1件: Amino acids---1件: 240
29Glycomacropeptide-based protein substitute----1件: 240
30Hepatocyte Transplant----1件: 240
31High oleic sunflower oil1件: Sunflower oil1件: D09006 --1件: 240
32HMI-103----1件: 240
33Immunosuppression----1件: 240
34KUVAN1件: Sapropterin1件: D08505 --1件: 240
35Kuvan 100mg soluble tablet1件: Sapropterin1件: D08505 --1件: 240
36KuvanTM Therapy----1件: 240
37Kuvan®1件: Sapropterin1件: D08505 --1件: 240
38Kuvan® (sapropterin dihydrochloride)1件: Sapropterin1件: D08505 --1件: 240
39Large Neutral Amino Acid Therapy----1件: 240
40Liver biopsy----4件: 93, 171, 231, 240
41Low calorie protein substitute----1件: 240
42Medikinet----1件: 240
43Microalgal oil----1件: 240
44Moxifloxacin1件: Moxifloxacin2件: D00874 ,
D08237 		--9件: 2, 6, 46, 49, 85, 86, 113, 240, 299
45N.a.----10件: 3, 6, 8, 13, 46, 96, 97, 240, 256, 271
46Nap----2件: 96, 240
47Other: Diet----1件: 240
48Other: Diet treatment----1件: 240
49Other: Liver Evaluation----1件: 240
50Other: Meal Challenge----1件: 240
51Other: Normal (control) diet----1件: 240
52Other: Phenylalanine (Phe)-restricted diet1件: Phenylalanine1件: D00021 --1件: 240
53Other: Routine----1件: 240
54Palynziq1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
55PEG----12件: 6, 13, 41, 46, 65, 78, 96, 97, 140, 144, 240, 299
56PEGVALIASE1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
57Phe----2件: 75, 240
58Phenoptin----2件: 124, 240
59Phenylalanine1件: Phenylalanine1件: D00021 --1件: 240
60Phenylalanine restricted diet1件: Phenylalanine1件: D00021 --1件: 240
61Phenylalanine-free protein substitute in tablet form (XPhe minis)1件: Phenylalanine1件: D00021 --1件: 240
62Phenylalanine-free protein substitute tablets1件: Phenylalanine1件: D00021 --1件: 240
63Phenylketonuria-type diet----1件: 240
64PKU Explore----1件: 240
65PKU express plus----1件: 240
66PKU Sphere----1件: 240
67PKU sphere liquid----1件: 240
68PKU Start----1件: 240
69PKU/TYR GMP Protein Substitute----1件: 240
70Protein intake----1件: 240
71PTC923----1件: 240
72Radiation: Preparative Radiation Therapy----1件: 240
73RAvPAL-PEG1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
74RAvPAL-PEG 0.001 mg/kg1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
75RAvPAL-PEG 0.003 mg/kg1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
76RAvPAL-PEG 0.01 mg/kg1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
77RAvPAL-PEG 0.03 mg/kg1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
78RAvPAL-PEG 0.1 mg/kg1件: Pegvaliase1件: D11077 1件: PAH 💬5件: Biosynthesis of amino acids, Folate biosynthesis, Metabolic pathways, Phenylalanine metabolism, Phenylalanine, tyrosine and tryptophan biosynthesis 💬1件: 240
79RTX----5件: 11, 46, 65, 222, 240
80RTX-134----1件: 240
81Sapropterin1件: Sapropterin1件: D08505 --3件: 86, 124, 240
82Sapropterin (Kuvan)1件: Sapropterin1件: D08505 --1件: 240
83SAPROPTERIN DIHYDROCHLORIDE1件: Sapropterin1件: D08505 --1件: 240
84Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin1件: Sapropterin1件: D08505 --1件: 240
85Sapropterin Dihydrocholoride1件: Sapropterin1件: D08505 --1件: 240
86SAPROPTERIN HYDROCHLORIDE1件: Sapropterin1件: D08505 --1件: 240
87Sapropterin, sapropterin dihydrochloride1件: Sapropterin1件: D08505 --1件: 240
88Sapropterin, sapropterin hydrochloride1件: Sapropterin1件: D08505 --1件: 240
89Sapropterindihydrochloride----1件: 240
90Saproterin dihydrochloride----1件: 240
91Sunflower oil1件: Sunflower oil1件: D09006 --3件: 46, 240, 299
92SYNB1618----1件: 240
93SYNB1934----1件: 240
94Synergy----1件: 240
95T1401----1件: 240
96Tetrahydrobiopterin1件: Sapropterin1件: D08505 --1件: 240
97Tetrahydrobiopterin (BH4)1件: Sapropterin1件: D08505 --1件: 240