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Mucopolysaccharidosis Type VI (Maroteaux-Lamy Syndrome): Defining and Measuring Functional Impacts in Pediatric Patients
source: Orphanet journal of rare diseases
year: 2021
authors: Leiro B,Phillips D,Duiker M,Harmatz P,Charles S
summary/abstract:Research about pediatric patients’ perspective on mucopolysaccharidosis type VI (MPS VI) and its impact on daily life is limited. We aimed to identify the disease concepts of interest that most impact function and day-to-day life of pediatric patients with MPS VI, and to consider clinical outcome assessments (COAs) that may potentially measure meaningful improvements in these concepts.
organization: Phillips Consulting, Chapel Hill, NC, USA. [email protected].DOI: 10.1186/s13023-021-02113-8
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