Showing results (1-10 of 595) with videos related to

Sort By:
Pageof 60
Pediatrics|August 3, 2007
Management guidelines for mucopolysaccharidosis VIRoberto Giugliani, Paul Harmatz, James E Wraith
Orphanet Journal of Rare Diseases|April 21, 2020
Assessing the impact of the five senses on quality of life in mucopolysaccharidosesRoberto Giugliani, Paul Harmatz, Shuan-Pei Lin, et al.
American Journal of Medical Genetics. Part A|April 26, 2014
Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey StudyRoberto Giugliani, Christina Lampe, Nathalie Guffon, et al.
Molecular Genetics and Metabolism Reports|May 31, 2018
Risks of long-term port use in enzyme replacement therapy for lysosomal storage disordersChristian J Hendriksz, Paul Harmatz, Roberto Giugliani, et al.
American Journal of Medical Genetics. Part A|December 7, 2017
Intrafamilial variability in the clinical manifestations of mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS)Can Ficicioglu, Roberto Giugliani, Paul Harmatz, et al.
Orphanet Journal of Rare Diseases|October 31, 2021
Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)Joseph Muenzer, Jaco Botha, Paul Harmatz, et al.
Pediatrics|February 5, 2008
Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)Rick Martin, Michael Beck, Christine Eng, et al.
Molecular Genetics and Metabolism|March 30, 2013
The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS)Simon A Jones, Rossella Parini, Paul Harmatz, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|January 15, 2011
Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome SurveyJoseph Muenzer, Michael Beck, Roberto Giugliani, et al.
American Journal of Medical Genetics. Part A|February 4, 2005
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)Stuart J Swiedler, Michael Beck, Manal Bajbouj, et al.
Pageof 60