检索结果 - Peter Slasor
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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 由 Christian J. Hendriksz, Barbara K. Burton, Thomas R. Fleming, Paul Harmatz, Derralynn Hughes, Simon Jones, Hsiang‐Yu Lin, Eugen Mengel, Maurizio Scarpa, Vassili Valayannopoulos, Roberto Giugliani, Peter Slasor, Debra L. Lounsbury, Wolfgang Dummer
出版 2014Artigo -
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Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study 由 Miriam Nickel, Alessandro Simonati, David Jacoby, Susanne Lezius, Dirk Kilian, Benjamin Van de Graaf, Odelya E. Pagovich, Barry E. Kosofsky, Kaleb Yohay, Matthew Downs, Peter Slasor, Temitayo Ajayi, Ronald G. Crystal, Alfried Kohlschütter, Dolan Sondhi, Angela Schulz
出版 2018Artigo -
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The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects 由 Paul Harmatz, Eugen Mengel, Roberto Giugliani, Vassili Valayannopoulos, Shuan-Pei Lin, Rossella Parini, Nathalie Guffon, Barbara K. Burton, Christian J. Hendriksz, John J. Mitchell, Ana María Martins, Simon Jones, Norberto Guelbert, Ashok Vellodi, Carla E. M. Hollak, Peter Slasor, Celeste Decker
出版 2013Artigo -
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Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study 由 Angela Schulz, Nicola Specchio, Emily de los Reyes, Paul Gissen, Miriam Nickel, Marina Trivisano, Shawn C. Aylward, Anupam Chakrapani, Christoph Schwering, Eva Wibbeler, Lena Marie Westermann, Douglas Ballon, Jonathan P. Dyke, Anu Cherukuri, Shailesh Bondade, Peter Slasor, Jessica Cohen Pfeffer
出版 2023Artigo -
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Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome 由 Paul Harmatz, Eugen Mengel, Roberto Giugliani, Vassili Valayannopoulos, Shuan-Pei Lin, Rossella Parini, Nathalie Guffon, Barbara K. Burton, Christian J. Hendriksz, John J. Mitchell, Ana María Martins, Simon Jones, Norberto Guelbert, Ashok Vellodi, Frits A. Wijburg, Kehu Yang, Peter Slasor, Celeste Decker
出版 2014Artigo -
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Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome 由 Christian J. Hendriksz, Rossella Parini, Moeenaldeen AlSayed, Julian Raiman, Roberto Giugliani, Martha Luz Solano Villarreal, John J. Mitchell, Barbara K. Burton, Norberto Guelbert, Fiona Stewart, Derralynn Hughes, Kenneth I. Berger, Peter Slasor, Robert Matousek, Elaina Jurecki, Adam J. Shaywitz, Paul Harmatz
出版 2016Artigo
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