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TITLE: Natural History and Galsulfase Treatment in Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)-10-Year Follow-Up of Patients who Previously Participated in an MPS VI Survey Study  Full Text
AUTHORS: Roberto Giugliani; Christina Lampe; Nathalie Guffon; David Ketteridge; Elisa Leao Teles; James E Wraith; Simon A Jones; Cheri Piscia Nichols; Ping Lin; Adrian Quartel; Paul Harmatz;
PUBLISHED: 2014, SOURCE: AMERICAN JOURNAL OF MEDICAL GENETICS PART A, VOLUME: 164A, ISSUE: 8
INDEXED IN: Scopus WOS CrossRef
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TITLE: Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)  Full Text
AUTHORS: Christian J Hendriksz; Roberto Giugliani; Paul Harmatz; Christina Lampe; Ana Maria Martins; Gregory M Pastores; Robert D Steiner; Elisa Leao Teles; Vassili Valayannopoulos;
PUBLISHED: 2013, SOURCE: JOURNAL OF INHERITED METABOLIC DISEASE, VOLUME: 36, ISSUE: 2
INDEXED IN: Scopus WOS CrossRef
13
TITLE: Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease  Full Text
AUTHORS: Maurizio Scarpa; Zsuzsanna Almassy; Michael Beck; Olaf Bodamer; Iain A Bruce; Linda De Meirleir; Nathalie Guffon; Encarna Guillen Navarro; Pauline Hensman; Simon Jones; Wolfgang Kamin; Christoph Kampmann; Christina Lampe; Christine A Lavery; Elisa Leao Teles; Bianca Link; Allan M Lund; Gunilla Malm; Susanne Pitz; Michael Rothera; Catherine Stewart; Anna Tylki Szymanska; Ans van der Ploeg; Robert Walker; Jiri Zeman; James E Wraith; ...More
PUBLISHED: 2011, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 6, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
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