Wraith, J.E.

patient disease mps ii study syndrome treatment therapy mutation enzyme hunter hunter syndrome change mucopolysaccharidosis type ii baseline enzyme replacement therapy mucopolysaccharidosi idursulfase month children infusion lysosomal diagnosis laronidase airway group hurler syndrome pompe trial rhgaa activity hurler level shire transplantation mps i genzyme corporation replacement hospital symptom assessment genet lysosomal storage diseases report manifestation antibody wraith pediatr airway obstruction university storage effect orphanet journal genzyme disorder 18 months support glycogen muscle swets blackwell 76157563 infantile-onset pompe disease motor mucopolysaccharidose deficiency pediatrics glycosaminoglycan family department improvement article bone marrow transplantation management development function death residue involvement pompe disease hgsnat control group lysosomal lumen changes