Mucopolysaccharidosis I under enzyme replacement therapy with laronidase—A mortality case with autopsy report

There is little information about MPS I‐related complications during laronidase therapy. We describe the first autopsy report of a young male MPS I patient who died of infection‐induced cardiopulmonary failure following 2 years of weekly treatment with laronidase.

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Bibliographische Detailangaben
Veröffentlicht in:	Journal of inherited metabolic disease 2005-12, Vol.28 (6), p.1146-1148
Hauptverfasser:	Lin, H.‐Y., Lin, S.‐P., Chuang, C.‐K., Chen, M.‐R., Chen, B.‐F., Wraith, J. E.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Autopsy Biological and medical sciences Carbohydrates (enzymatic deficiencies). Glycogenosis Errors of metabolism Fatal Outcome Glycosaminoglycans - metabolism Heart Failure Heparitin Sulfate - metabolism Humans Iduronidase - chemistry Iduronidase - deficiency Iduronidase - therapeutic use Infection - mortality Male Medical genetics Medical sciences Metabolic diseases Miscellaneous Mucopolysaccharidosis I - drug therapy Mucopolysaccharidosis I - mortality Pharmaceutical Preparations Public health. Hygiene Public health. Hygiene-occupational medicine Recombinant Proteins - therapeutic use
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Beschreibung
Zusammenfassung:	There is little information about MPS I‐related complications during laronidase therapy. We describe the first autopsy report of a young male MPS I patient who died of infection‐induced cardiopulmonary failure following 2 years of weekly treatment with laronidase.
ISSN:	0141-8955 1573-2665
DOI:	10.1007/s10545-005-0211-x