Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy

Treatments for Mucopolysaccharidosis IVA (MPS IVA, also called Morquio A syndrome) by enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) are limited in efficacy depending on the age of initiation and clinical phenotype. Thus, this study aims to assess the effects of treatments on MPS IVA patients compared to untreated MPS IVA patients and an age-matched control group.

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