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.

[1]  R. Giugliani,et al.  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 N-Acetylgalactosamine 4-Sulfatase , 2005, Pediatrics.

[2]  R. Giugliani,et al.  Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux–Lamy syndrome) , 2005, American journal of medical genetics. Part A.

[3]  E. Miebach Enzyme replacement therapy in mucopolysaccharidosis type I , 2005, Acta paediatrica (Oslo, Norway : 1992). Supplement.

[4]  Joseph Muenzer,et al.  Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). , 2004, The Journal of pediatrics.

[5]  P. Kaplan,et al.  Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). , 2004, The Journal of pediatrics.

[6]  J. Lemontt,et al.  Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy. , 2003, Molecular genetics and metabolism.

[7]  A. Branzi,et al.  The new clinical trials on pharmacological treatment in pulmonary arterial hypertension , 2002, European Respiratory Journal.

[8]  V. Hasselblad,et al.  Prognostic usefulness of the six-minute walk in patients with advanced congestive heart failure secondary to ischemic or nonischemic cardiomyopathy. , 2001, The American journal of cardiology.

[9]  M. Fujita,et al.  Clinical correlates and prognostic significance of six-minute walk test in patients with primary pulmonary hypertension. Comparison with cardiopulmonary exercise testing. , 2000, American journal of respiratory and critical care medicine.

[10]  D A Redelmeier,et al.  Interpreting small differences in functional status: the Six Minute Walk test in chronic lung disease patients. , 1997, American journal of respiratory and critical care medicine.

[11]  D. Brooks,et al.  Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome. , 1996, The Journal of clinical investigation.

[12]  John L. Hankinson,et al.  Standardization of Spirometry, 1994 Update. American Thoracic Society. , 1995, American journal of respiratory and critical care medicine.

[13]  C. Hornung,et al.  Determinants of achievement in stair climbing as an exercise test. , 1994, Military Medicine.

[14]  William J. Rogers,et al.  Prediction of Mortality and Morbidity With a 6-Minute Walk Test in Patients With Left Ventricular Dysfunction , 1993 .

[15]  Bittner,et al.  Prediction of mortality and morbidity with a 6-minute walk test in patients with left ventricular dysfunction. SOLVD Investigators. , 1993, JAMA.

[16]  M. Kaiser-Kupfer,et al.  Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses , 1992, Journal of Inherited Metabolic Disease.

[17]  J. Neglia,et al.  Diagnostic test for mucopolysaccharidosis. I. Direct method for quantifying excessive urinary glycosaminoglycan excretion. , 1989, Clinical chemistry.

[18]  C. Hornung,et al.  Stair climbing as an indicator of pulmonary function. , 1987, Chest.

[19]  D. Snover,et al.  Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Biochemical and clinical status 24 months after transplantation. , 1984, The New England journal of medicine.

[20]  Charles R.scriver,et al.  The Metabolic basis of inherited disease , 1989 .