Published September 10, 2019
| Version v1
Journal article
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LA MUCOPOLYSACCHARIDOSE DE TYPE I: EFFICACITE DU TRAITEMENT ENZYMATIQUE SUBSTITUTIF PAR LA LARONIDASE CHEZ UN CAS.
Authors/Creators
- 1. Laboratoire de Biotechnologies et Valorisation des Ressources Naturelles, d?partement de biologie, universit? IBN ZOHR, Agadir, Maroc.
- 2. Laboratoire de Biologie Cellulaire et G?n?tique Mol?culaire d?partement de biologie, universit? IBN ZOHR, Agadir, Maroc.
- 3. Cabinet p?diatrique, Agadir, Maroc.
Description
Mucopolusaccharidosis Type I (MPS I) is a lysosomal storage disease due to α-L-iduronidase deficiency. Currently, the disease is treated buy the recombinant human α-L-iduronidase (laronidase). This study evaluates the clinical outcome and quality of life, after two years of traitement by laronidase in a 10 years aged boy, late diagnosed at age 8 with Hurler-Scheie syndrome. The results of the ETS are encouraging. Indeed, this treatment was benefic since it has improved his quality of life; however it have not cure his disease.