Maurizio Scarpa

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 Döederlein Schwartz et al. · The Journal of Pediatrics · 2006 · 361 citations

Translational Medicine, Phase 3, Enzyme Replacement Therapy +7

ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo

Stefano Ferrari, Enrico Moro, Andrea Pettenazzo et al. · Gene Therapy · 1997 · 252 citations

In Vivo Gene Therapy, Exgen 500, Bronchial Neoplasm +8

A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

Barbara K. Burton, Manisha Balwani, François Feillet et al. · New England Journal of Medicine · 2015 · 238 citations · Full text

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease

Maurizio Scarpa, Zsuzsanna Almássy, Michael Beck et al. · Orphanet Journal of Rare Diseases · 2011 · 223 citations · Full text

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

Paul Harmatz, Roberto Giugliani, Ida Vanessa Döederlein Schwartz et al. · Molecular Genetics and Metabolism · 2008 · 215 citations

Translational Medicine, Enzyme Replacement Therapy, Pathology +4