Clara Sá-Miranda

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

Prevalence of lysosomal storage diseases in Portugal

Rui Pinto, Carla Caseiro, Manuela Nascimento de Lemos et al. · European Journal of Human Genetics · 2003 · 347 citations · Full text

Lysosomal Storage Diseases, Mendelian Disorder, Genetic Disorder +7

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

Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement Therapy of Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): Results After 48 Weeks in a Phase 2 Open-Label Clinical Study of Recombinant Human <i>N</i>-Acetylgalactosamine 4-Sulfatase

Paul Harmatz, David Ketteridge, Roberto Giugliani et al. · PEDIATRICS · 2005 · 213 citations

Ubiquitination of Mammalian Pex5p, the Peroxisomal Import Receptor

Andreia F. Carvalho, Manuel P. Pinto, Cláudia P. Grou et al. · Journal of Biological Chemistry · 2007 · 172 citations · Full text