M
Maurizio Scarpa
Researcher at University of Padua
Publications - 202
Citations - 6979
Maurizio Scarpa is an academic researcher from University of Padua. The author has contributed to research in topics: Enzyme replacement therapy & Medicine. The author has an hindex of 39, co-authored 167 publications receiving 5927 citations. Previous affiliations of Maurizio Scarpa include Casa Sollievo della Sofferenza & European Bioinformatics Institute.
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Journal ArticleDOI
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
J. Edmond Wraith,Maurizio Scarpa,Michael Beck,Olaf Bodamer,Linda De Meirleir,Nathalie Guffon,Allan M. Lund,Gunilla Malm,Ans T. van der Ploeg,Jiri Zeman +9 more
TL;DR: ERT with idursulfase has the potential to benefit many patients with MPS II, especially if started early in the course of the disease, and recommendations for the use of ERT are provided.
Journal ArticleDOI
Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study.
Paul Harmatz,Roberto Giugliani,Ida Vanessa Doederlein Schwartz,Nathalie Guffon,Elisa Leão Teles,M. Clara Sá Miranda,J. Edmond Wraith,Michael Beck,Laila Arash,Maurizio Scarpa,Zi-Fan Yu,Janet Wittes,Kenneth I. Berger,Mary S. Newman,Ann M. Lowe,Emil D. Kakkis,Stuart J. Swiedler +16 more
TL;DR: rhASB significantly improves endurance, reduces GAG, and has an acceptable safety profile, which is generally safe and well tolerated.
Journal ArticleDOI
ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo.
Stefano Ferrari,Enrico Moro,Andrea Pettenazzo,Jean-Paul Behr,Franco Zacchello,Maurizio Scarpa +5 more
TL;DR: In vivo ExGen 500 was able to mediate gene transfer into both newborn and adult rabbit lungs with comparable efficiencies, and within 1 week after instillation, transgene expression decreased by two orders of magnitude.
Journal ArticleDOI
Immunologic Privilege in the Central Nervous System and the Blood–Brain Barrier
Leslie L. Muldoon,Jorge I. Alvarez,David J. Begley,Ruben J. Boado,Gregory J. del Zoppo,Nancy D. Doolittle,Britta Engelhardt,John M. Hallenbeck,Russell R. Lonser,John R. Ohlfest,Alexandre Prat,Maurizio Scarpa,Richard J. Smeyne,Lester R. Drewes,Edward A. Neuwelt +14 more
TL;DR: The goals of this review are to outline key scientific advances and the status of the science central to both the neuroinflammation and CNS barriers fields, and highlight the opportunities and priorities in advancing brain barriers research in the context of the larger immunology and neuroscience disciplines.
Journal ArticleDOI
Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management
Elizabeth A. Braunlin,Paul Harmatz,Maurizio Scarpa,Beatriz Furlanetto,Christoph Kampmann,James P. Loehr,Katherine P. Ponder,William C. Roberts,Howard M. Rosenfeld,Roberto Giugliani +9 more
TL;DR: Cardiac involvement has been reported in all MPS syndromes and is a common and early feature, particularly for those with MPS I, II, and VI, although ERT within the first few months of life may prevent valve involvement, a fact that emphasizes the importance of early diagnosis and treatment in MPS.