Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI

The metabolic and molecular bases of inherited disease

Ying-Tsong Chen · Medical Entomology and Zoology · 2001 · 6.9K citations

Disease Mechanism, Genetic Disorder, Genetics +6

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

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Paul Harmatz, Chester B. Whitley, Lewis Waber et al. · The Journal of Pediatrics · 2004 · 273 citations

Rare Diseases, Enzyme Replacement Therapy, Genetic Disorder +5

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

Mechanism of Glycosaminoglycan-Mediated Bone and Joint Disease

Calogera M. Simonaro, Marina D’Angelo, Xingxuan He et al. · American Journal Of Pathology · 2007 · 201 citations · Full text

Bone Disease, Rheumatology, Osteoarthritis +6