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Ataluren

PTC124,PTC-124,PTC 124

PTC124 (Ataluren)选择性诱导核糖体通读，不影响正常的终止密码子，EC50为0.1 μM，可以治疗无义突变（如CFTR无义突变引起的囊性纤维病）造成的遗传性疾病。

目录号
EY0744
EY0744
EY0744
EY0744
EY0744
纯度
99.64%
99.64%
99.64%
99.64%
99.64%
规格
1 mg
5 mg
10 mg
50 mg
100 mg
原价
140
295
486
1400
2000
售价
140
295
486
1400
2000
库存
现货
现货
现货
现货
现货
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订购
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  • 生物活性

    Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). Phase 3.

  • 体外研究

  • 体内研究

  • 激酶实验

  • 细胞实验

  • 动物实验

    ~60 mg/kg/day皮下注射或口服处理

  • 不同实验动物依据体表面积的等效剂量转换表（数据来源于FDA指南）

    动物 A (mg/kg) = 动物 B (mg/kg)×动物 B的Km系数/动物 A的Km系数


    例如，已知某工具药用于小鼠的剂量为88 mg/kg , 则用于大鼠的剂量换算方法：将88 mg/kg 乘以小鼠的Km系数（3），再除以大鼠的Km系数（6），得到该药物用于大鼠的等效剂量44 mg/kg。

  • 参考文献

    [1] Welch EM, et al. Nature, 2007, 447(7140), 87-91.
    [2] Auld, D.S., et al. 2009. Proc. Natl. Acad. Sci. U.S.A. 106: 3585-3590.
    [3] Du, M., et al. 2008. Proc. Natl. Acad. Sci. U.S.A. 105: 2064-2069.
    [4] Kerem, E., et al. 2008. Lancet. 372: 719-727.

    分子式
    C15H9FN2O3    		 分子量
    284.24    		 CAS号
    775304-57-9    		 储存方式
    ﹣20 ℃冷藏长期储存。冰袋运输
    溶剂（常温）
    		 DMSO
    ≥55 mg/mL    		 Water
    <1 mg/mL    		 Ethanol
    <1 mg/mL

    体内溶解度

  • Clinical Trial Information ( data from http://clinicaltrials.gov )

    NCT Number Conditions Interventions Sponsor/Collaborators Phases Start Date Last Updated
    NCT02456103 Cystic Fibrosis Drug: Ataluren PTC Therapeutics Phase 3 2015-07-01 2015-05-26
    NCT02090959 Muscular Dystrophy, Duchenne|Muscular Dystrophies|Muscular Disorders, Atrophic|Muscular Diseases|Musculoskeletal Diseases|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases, X-Linked|Genetic Diseases, Inborn Drug: Ataluren PTC Therapeutics Phase 3 2014-03-01 2014-10-15
    NCT02758626 Epilepsy Drug: ataluren|Drug: Placebo New York University School of Medicine|PTC Therapeutics Phase 2 2016-11-01 2016-11-22
    NCT02819557 Duchenne Muscular Dystrophy Drug: Ataluren PTC Therapeutics Phase 2 2016-06-01 2016-12-15
    NCT01557400 Duchenne Muscular Dystrophy|Becker Muscular Dystrophy|Dystrophinopathy Drug: Ataluren PTC Therapeutics Phase 3 2012-05-01 2016-07-26
    NCT01140451 Cystic Fibrosis Drug: Ataluren (PTC124) PTC Therapeutics|Cystic Fibrosis Foundation Therapeutics Phase 3 2010-08-01 2016-03-01
    NCT00847379 Duchenne Muscular Dystrophy|Becker Muscular Dystrophy Drug: Ataluren (PTC124) PTC Therapeutics|Genzyme, a Sanofi Company Phase 2|Phase 3 2009-01-01 2013-08-28
    NCT01009294 Duchenne Muscular Dystrophy|Becker Muscular Dystrophy Drug: Ataluren (PTC124) PTC Therapeutics|Genzyme, a Sanofi Company Phase 2 2009-11-01 2013-08-28
    NCT00759876 Duchenne Muscular Dystrophy Drug: Ataluren PTC Therapeutics|Genzyme, a Sanofi Company Phase 2 2008-07-01 2013-08-13
    NCT02107859 Cystic Fibrosis Drug: Ataluren (PTC124) PTC Therapeutics Phase 3 2014-05-01 2016-05-06
    NCT01141075 Amino Acid Metabolism, Inborn Errors Drug: Ataluren (PTC124) PTC Therapeutics|Genzyme, a Sanofi Company Phase 2 2010-06-01 2011-10-31
    NCT01826487 Muscular Dystrophy, Duchenne|Muscular Dystrophies|Muscular Disorders, Atrophic|Muscular Diseases|Musculoskeletal Diseases|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases, X-Linked|Genetic Diseases, Inborn Drug: Ataluren|Drug: Placebo PTC Therapeutics Phase 3 2013-03-01 2016-08-02
    NCT02647359 Aniridia Drug: Ataluren|Drug: Placebo PTC Therapeutics Phase 2 2016-01-01 2016-12-13
    NCT00803205 Cystic Fibrosis Drug: Ataluren (PTC124)|Drug: Placebo PTC Therapeutics|Cystic Fibrosis Foundation Therapeutics Phase 3 2009-07-01 2013-08-14
    NCT01247207 Duchenne Muscular Dystrophy|Becker Muscular Dystrophy Drug: Ataluren PTC Therapeutics Phase 3 2010-11-01 2016-07-20
    NCT00947193 Hemophilia A|Hemophilia B Drug: Ataluren (PTC124) PTC Therapeutics|Genzyme, a Sanofi Company Phase 2 2009-08-01 2011-10-31
    NCT02409004 Healthy Drug: Ataluren|Drug: Rifampin PTC Therapeutics Phase 1 2015-03-01 2015-03-31
    NCT00592553 Duchenne Muscular Dystrophy|Becker Muscular Dystrophy Drug: PTC124|Drug: PTC124|Drug: PTC124 PTC Therapeutics Phase 2|Phase 3 2008-02-01 2013-09-06
    NCT00264888 Duchenne Muscular Dystrophy Drug: PTC124 PTC Therapeutics|Muscular Dystrophy Association Phase 2 2005-12-01 2009-01-09
    NCT00351078 Cystic Fibrosis Drug: PTC124 PTC Therapeutics Phase 2 2006-12-01 2007-10-16
    NCT00234663 Cystic Fibrosis Drug: PTC124 PTC Therapeutics|Cystic Fibrosis Foundation Therapeutics|FDA Office of Orphan Products Development Phase 2 2005-09-01 2009-01-09
    NCT00458341 Cystic Fibrosis Drug: PTC124 PTC Therapeutics Phase 2 2007-03-01 2009-04-01
    NCT00237380 Cystic Fibrosis Drug: PTC124 PTC Therapeutics Phase 2 2005-11-01 2009-01-09

    注：以上所有数据均来自公开文献，并不保证对所有实验均有效，数据仅供参考。

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