Gasser cell: A biomarker of response to enzyme replacement therapy in patients with mucopolysaccharidosis type VI
نویسندگان
چکیده
The mucopolysaccharidosis (MPS) type VI is a rare lysosomal storage disease presenting leukocyte inclusions (Alder-Reilly anomaly) and lymphocytes with metachromatic inclusion surrounded by clear spaces, Gasser cells. Currently, an enzyme replacement therapy (ERT) galsulfase used to treat MPS VI. This study evaluated 14 patients performed cell counts before after six months from the beginning of ERT. It was observed average 12.7% cells per patient, found complete disappearance, proving be effective biomarker response
منابع مشابه
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ژورنال
عنوان ژورنال: Research, Society and Development
سال: 2021
ISSN: ['2525-3409']
DOI: https://doi.org/10.33448/rsd-v10i5.14726