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Mucopolysaccharidoses (MPS) are rare genetic diseases caused by the deficiency of one of the lysosomal enzymes involved in the glycosaminoglycan (GAG) breakdown pathway. This metabolic block leads to the accumulation of GAG in various organs and tissues of the affected patients, resulting in a multisystemic clinical picture, sometimes including cognitive impairment. Until the beginning of the XXI century, treatment was mainly supportive. Bone marrow transplantation improved the natural course of the disease in some types of MPS, but the morbidity and mortality restricted its use to selected cases. The identification of the genes involved, the new molecular biology tools and the availability of animal models made it possible to develop specific enzyme replacement therapies (ERT) for these diseases. At present, a great number of Brazilian medical centers from all regions of the country have experience with ERT for MPS I, II, and VI, acquired not only through patient treatment but also in clinical trials. Taking the three types of MPS together, over 200 patients have been treated with ERT in our country. This document summarizes the experience of the professionals involved, along with the data available in the international literature, bringing together and harmonizing the information available on the management of these severe and progressive diseases, thus disclosing new prospects for Brazilian patients affected by these conditions.

Index code	Element
ti	article title
au	author
kw	article keywords
subject	subject (title words, abstract and keywords)
ab	abstract
ta	journal short title (e.g. Cad. Saúde Pública)
journal_title	journal full title (e.g. Cadernos de Saúde Pública)
la	publication language code (e.g. pt - Portuguese, es - Spanish)
type	document type
pid	publication identifier
publication_year	publication year of publication
sponsor	sponsor
aff_country	country code of the author's affiliation
aff_institution	author affiliation institution
volume	article volume
issue	article issue
elocation	elocation
doi	DOI number
issn	journal ISSN
in	SciELO colection code (e.g. scl - Brasil, col - Colômbia)
use_license	article usage license code

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