Laronidase (Aldurazyme): enzyme replacement therapy for mucopolysaccharidosis type I.
BACKGROUND
Laronidase (Aldurazyme) is a recombinant formulation of alpha-L-iduronidase, the enzyme deficient in mucopolysaccharidosis type I (MPS-I); a disorder associated with skeletal dysplasia, restricted joint movement, short stature, obstructive pulmonary disease, cardiac valvular problems and cognitive impairment (in the severe and intermediate variants).


OBJECTIVE
To describe MPS-I and review data on the safety and efficacy of laronidase.


RESULTS
Laronidase is safe and effective in stabilizing or improving pulmonary function and physical endurance. As intravenously administered enzyme is unable to correct CNS disease, hematopoietic stem cell transplantation remains the primary treatment for Hurler's syndrome despite the morbidity and mortality risks.


CONCLUSIONS
Palliative care remains part of the treatment. Long-term studies are required to ascertain the effect of enzyme therapy on survival and its effectiveness in modifying the disease course and reducing morbidity. Intrathecal administration is under investigation for patients with signs of cord compression secondary to glycosaminoglycan accumulation within the dura matter. The cost of therapy remains a concern.
New York University School of Medicine, 403 East 34th Street, 2nd Floor, New York, NY 10016, USA. gregory.pastores@med.nyu.edu
Gregory M
GM
Pastores
D000293:Adolescent; D000328:Adult; D000818:Animals; D001691:Biological Therapy / Q000379:methods*; D001921:Brain / Q000378:metabolism; D002648:Child; D002986:Clinical Trials as Topic; D004285:Dogs; D006025:Glycosaminoglycans / Q000378:metabolism; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D007068:Iduronidase / Q000627:therapeutic use*; D008954:Models, Biological; D009083:Mucopolysaccharidoses / Q000628:therapy*; D004364:Pharmaceutical Preparations
D006025:Glycosaminoglycans; D004364:Pharmaceutical Preparations; D007068:Iduronidase