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Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome)

Fabiano de Oliveira Poswar1,2,3,4, Johanna Henriques Nehm1, Francyne Kubaski5

  • 1Clinical Research Group in Medical Genetics, Clinical Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.

Therapeutics and Clinical Risk Management
|December 29, 2022
PubMed

Keywords:
Sly syndromeenzyme replacement therapygene therapyhematopoietic stem cell transplantationlysosomal disordersmucopolysaccharidosis type VII

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