000 01277 a2200373 4500
005 20250516152749.0
264 0 _c20140122
008 201401s 0 0 eng d
022 _a1096-7206
024 7 _a10.1016/j.ymgme.2013.04.023
_2doi
040 _aNLM
_beng
_cNLM
100 1 _aLaraway, Sarah
245 0 0 _aDoes early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings.
_h[electronic resource]
260 _bMolecular genetics and metabolism
_cJul 2013
300 _a315-6 p.
_bdigital
500 _aPublication Type: Case Reports; Journal Article
650 0 4 _aChild
650 0 4 _aChild, Preschool
650 0 4 _aEnzyme Replacement Therapy
650 0 4 _aFemale
650 0 4 _aHumans
650 0 4 _aIduronidase
_xtherapeutic use
650 0 4 _aInfant
650 0 4 _aMucopolysaccharidosis I
_xdrug therapy
650 0 4 _aPhenotype
650 0 4 _aSiblings
650 0 4 _aTreatment Outcome
700 1 _aBreen, Catherine
700 1 _aMercer, Jean
700 1 _aJones, Simon
700 1 _aWraith, James E
773 0 _tMolecular genetics and metabolism
_gvol. 109
_gno. 3
_gp. 315-6
856 4 0 _uhttps://doi.org/10.1016/j.ymgme.2013.04.023
_zAvailable from publisher's website
999 _c22793901
_d22793901