Disease burden, management patterns and multidisciplinary clinical approaches for patients with MPS IVA and VI in selected Latin American Countries.

ABSTRACT:

Background

There is a paucity of real-world epidemiological data on patients with mucopolysaccharidoses (MPS) in Latin America. This real-world study assessed the disease burden, management patterns and multidisciplinary clinical approaches for MPS-IVA and MPS-VI patients in Latin America (Colombia, Ecuador, Mexico, Peru).

Methods

Data were collected from physicians/specialists experienced in treating MPS patients between April-June 2020, via an online patient-diary survey.

Results

Overall, 29 physicians/specialists participated in this study. Data from 98 patients were analyzed (MPS-IVA, 71 patients and MPS-VI, 27 patients). Mean age for MPS-IVA patients was 17.5 years and for MPS-VI patients was 11.6 years, and the majority were females (52% and 78%, respectively).

SUBMITTER: Solano VM 

PROVIDER: S-EPMC8170147 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature