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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
6BH4----5件: 51, 86, 124, 240, 299
7Blood samples----9件: 13, 41, 51, 61, 97, 227, 240, 271, 280
8BMN 165----1件: 240
9BMN 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
10BMN 307----1件: 240
11BMN165 20mg/day----1件: 240
12BMN165 40mg/day----1件: 240
13CDX----2件: 222, 240
14CDX 6114----1件: 240
15CNSA-001----1件: 240
16Cohort 1 0.225g----1件: 240
17Cohort 2 0.75g----1件: 240
18Cohort 3 2.25 g----1件: 240
19Corn1件: Corn---2件: 46, 240
20Corn/soy oil1件: Corn---1件: 240
21Diet----4件: 86, 97, 240, 299
22Dietary Supplement for PKU patients----1件: 240
23Docosahexaenoic acid1件: Doconexent---7件: 6, 49, 90, 94, 240, 299, 301
24Free amino acids intake1件: Amino acids---1件: 240
25Glycomacropeptide-based protein substitute----1件: 240
26HMI-102----1件: 240
27HMI-103----1件: 240
28Immunosuppression----1件: 240
29KUVAN1件: Sapropterin1件: D08505 --1件: 240
30Kuvan 100mg soluble tablet1件: Sapropterin1件: D08505 --1件: 240
31KuvanTM Therapy----1件: 240
32Kuvan®1件: Sapropterin1件: D08505 --1件: 240
33Kuvan® (sapropterin dihydrochloride)1件: Sapropterin1件: D08505 --1件: 240
34Large Neutral Amino Acid Therapy----1件: 240
35Low calorie protein substitute----1件: 240
36Medikinet----1件: 240
37Moxifloxacin1件: Moxifloxacin1件: D08237 --9件: 2, 6, 46, 49, 85, 86, 113, 240, 299
38N.a.----10件: 3, 6, 8, 13, 46, 96, 97, 240, 256, 271
39Nap----2件: 96, 240
40Palynziq1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
41PEG----15件: 6, 13, 41, 46, 60, 65, 78, 96, 97, 140, 144, 240, 246, 299, 337
42PEGVALIASE1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
43Pegvaliase-Pqpz1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
44Phenoptin----2件: 124, 240
45Phenylalanine1件: Phenylalanine1件: D00021 --1件: 240
46Phenylalanine restricted diet1件: Phenylalanine1件: D00021 --1件: 240
47Phenylalanine-free protein substitute in tablet form (XPhe minis)1件: Phenylalanine1件: D00021 --1件: 240
48Phenylalanine-free protein substitute tablets1件: Phenylalanine1件: D00021 --1件: 240
49Phenylketonuria-type diet----1件: 240
50PKU Explore----1件: 240
51PKU express plus----1件: 240
52PKU Sphere----1件: 240
53PKU sphere liquid----1件: 240
54PKU Start----1件: 240
55PKU/TYR GMP Protein Substitute----1件: 240
56Protein intake----1件: 240
57PTC923----1件: 240
58RAvPAL-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
59RAvPAL-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
60RAvPAL-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
61RAvPAL-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
62RAvPAL-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
63RAvPAL-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
64RTX----5件: 11, 46, 65, 222, 240
65RTX-134----1件: 240
66Sapropterin1件: Sapropterin1件: D08505 --3件: 86, 124, 240
67Sapropterin (Kuvan)1件: Sapropterin1件: D08505 --1件: 240
68SAPROPTERIN DIHYDROCHLORIDE1件: Sapropterin1件: D08505 --1件: 240
69Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin1件: Sapropterin1件: D08505 --1件: 240
70Sapropterin Dihydrocholoride1件: Sapropterin1件: D08505 --1件: 240
71SAPROPTERIN HYDROCHLORIDE1件: Sapropterin1件: D08505 --1件: 240
72Sapropterin, sapropterin dihydrochloride1件: Sapropterin1件: D08505 --1件: 240
73Sapropterin, sapropterin hydrochloride1件: Sapropterin1件: D08505 --1件: 240
74Sapropterindihydrochloride----1件: 240
75Saproterin dihydrochloride----1件: 240
76SYNB1618----1件: 240
77SYNB1934----1件: 240
78Synergy----1件: 240
79T1401----1件: 240
80Tetrahydrobiopterin1件: Sapropterin1件: D08505 --1件: 240
81Tetrahydrobiopterin (BH4)1件: Sapropterin1件: D08505 --1件: 240