Pharmacological and clinical profile of nitisinone (Orfadin<sup>®</sup> Capsules): a therapeutic agent for hereditary tyrosinemia type 1

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Clinical utility of nitisinone for the treatment of hereditary tyrosinemia type-1 (HT-1)

Medical therapy for hereditary hepatorenal tyrosinemia (hereditary tyrosinemia type 1, HT-1) with nitisinone was discovered incidentally, and is a by-product of agrochemistry. It blocks the catabolic pathway of tyrosine, thereby leading to a reduction in the accumulation of toxic metabolites in HT-1. It has to be combined with a low-protein diet supplemented with amino acid mixtures devoid of t...

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[Pharmacological and clinical profile of bixalomer (Kiklin(®) capsules): a new therapeutic agent for hyperphosphatemia].

キーワード:ビキサロマー,リン酸結合性ポリマー,高リン血症,膨潤,消化器副作用 アステラス製薬株式会社 研究本部 薬理研究所(〒305 -8585 茨城県つくば市御幸が丘 21) E-mail: [email protected] 原稿受領日:2013 年 1 月 18 日,依頼原稿 Title: Pharmacological and clinical profile of bixalomer (Kiklin capsules): a new therapeutic agent for hyperphosphatemia Author: Keiichi Taniguchi, Hirotoshi Kakuta, Yuichi Tomura, Seiji Kaku, Wataru Uchida 要約:ビキサロマー(キックリンカプセル)はリン 酸吸着ポリマーであ...

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Goldenhar syndrome and hereditary tyrosinemia type 1.

We report a case of Goldenhar syndrome and hereditary tyrosinemia type 1 (HTT1), to our knowledge an association not previously described. This case further increases the diversity of observations and clinical descriptions of patients with this complex syndrome. We discuss pathogenetic aspects, and demonstrate further evidence of the effectiveness of 2-(2-nitro-4-trifluoromethyl benzoyl)-1,3-cy...

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Clinical, biochemical, and genetic analysis of a Korean neonate with hereditary tyrosinemia type 1.

BACKGROUND Hereditary tyrosinemia type 1 (HT1; MIM 276700) is caused by mutations in the fumarylaceto-acetate hydrolase (FAH) gene, and is the most severe disorder associated with the tyrosine catabolic pathway. HT1 is a very rare disorder and no genetically confirmed case of HT1 in Korea has yet been reported. In this study, we present a Korean neonate with clinical and biochemical features of...

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ژورنال

عنوان ژورنال: Folia Pharmacologica Japonica

سال: 2015

ISSN: 0015-5691,1347-8397

DOI: 10.1254/fpj.146.342