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Sources of variation in estimates of Duchenne and Becker muscular dystrophy prevalence in the United States.
Assessing the impact of the five senses on quality of life in mucopolysaccharidoses.
Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance.
Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance.
Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry.
Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.