Parental perception of treatment options for mucopolysaccharidosis: a survey to bridge the gap for personalized medicine.

ABSTRACT:

Background

Mucopolysaccharidosis (MPS) are a group of lysosomal storage diseases with substantial unmet medical needs-for both patients and caregivers. Approved therapies are limited, and the perception of investigative ones remains enigmatic.

Method

Using an innovative survey concept based on the discrete choice experiment method (DEC) with neuronopathic and non-neuronopathic patient scenarios, we aimed to evaluate how parents of children with MPS perceive different approved and innovative therapies. The questionnaire was distributed via patient organizations in Germany, Switzerland, and Austria.

Results

Most likely parents would choose an approach to repurposed treatments for their child (neuronopathic: 82%, 14/17 and non-neuronopathic: 94%, 16/17), followed by enzy

SUBMITTER: Wiesinger AM 

PROVIDER: S-EPMC11762465 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature