Works matching IS 01418955 AND DT 2014 AND VI 37 AND IP 6

Results: 17
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    Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.

    Published in:
    Journal of Inherited Metabolic Disease, 2014, v. 37, n. 6, p. 979, doi. 10.1007/s10545-014-9715-6
    By:
    • Hendriksz, Christian;
    • Burton, Barbara;
    • Fleming, Thomas;
    • Harmatz, Paul;
    • Hughes, Derralynn;
    • Jones, Simon;
    • Lin, Shuan-Pei;
    • Mengel, Eugen;
    • Scarpa, Maurizio;
    • Valayannopoulos, Vassili;
    • Giugliani, Roberto;
    • Slasor, Peter;
    • Lounsbury, Debra;
    • Dummer, Wolfgang
    Publication type:
    Article
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    Erratum to: Clinical, biochemical and molecular analysis of 13 Japanese patients with β-ureidopropionase deficiency demonstrates high prevalence of the c.977G > A (p.R326Q) mutation.

    Published in:
    2014
    By:
    • Nakajima, Yoko;
    • Meijer, Judith;
    • Dobritzsch, Doreen;
    • Ito, Tetsuya;
    • Meinsma, Rutger;
    • Abeling, Nico;
    • Roelofsen, Jeroen;
    • Zoetekouw, Lida;
    • Watanabe, Yoriko;
    • Tashiro, Kyoko;
    • Lee, Tomoko;
    • Takeshima, Yasuhiro;
    • Mitsubuchi, Hiroshi;
    • Yoneyama, Akira;
    • Ohta, Kazuhide;
    • Eto, Kaoru;
    • Saito, Kayoko;
    • Kuhara, Tomiko;
    • Kuilenburg, André
    Publication type:
    Erratum