Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.

ABSTRACT:

Introduction

Several new treatment modalities are being developed for lysosomal storage disorders (LSDs), including gene therapy. As the currently available treatment options and their influence on disease progression differ greatly within the spectrum of LSDs, willingness to undergo gene therapy might vary among patients with LSDs and/or their representatives. The width of the LSD spectrum is illustrated by the differences between type 1 Gaucher disease, Fabry disease and Mucopolysaccharidosis type III (MPS III). For type 1 Gaucher and Fabry disease several therapies are available, resulting in a near normal or improved, but individually varying, prognosis. No treatment options are available for MPS III.

Aim

To identify factors influencing patients' and/or their representat

SUBMITTER: Eskes ECB 

PROVIDER: S-EPMC9587648 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature