Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings.

Laraway, Sarah

Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings. [electronic resource] - Molecular genetics and metabolism Jul 2013 - 315-6 p. digital

Publication Type: Case Reports; Journal Article

1096-7206

10.1016/j.ymgme.2013.04.023 doi


Child
Child, Preschool
Enzyme Replacement Therapy
Female
Humans
Iduronidase--therapeutic use
Infant
Mucopolysaccharidosis I--drug therapy
Phenotype
Siblings
Treatment Outcome