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
Christian J. Hendriksz(Birmingham Children's Hospital), Wolfgang Dummer(BioMarin (United States)), Maurizio Scarpa(University of Udine), Hsiang‐Yu Lin(Mackay Memorial Hospital), Peter Slasor(BioMarin (United States)), Vassili Valayannopoulos(Regeneron (United States)), Simon Jones(Manchester Academic Health Science Centre), Eugen Mengel(University of Hohenheim), Debra L. Lounsbury(BioMarin (United States)), Thomas R. Fleming(Cornell University), Roberto Giugliani(Instituto de Biologia Molecular e Celular), Paul Harmatz(Royal Manchester Children's Hospital), Barbara K. Burton(Women & Children's Hospital of Buffalo), Derralynn Hughes(Royal Free London NHS Foundation Trust)
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