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

Eline C B EskesCathrien R L BeishuizenEleonore M CorazollaTessa van MiddelaarMarion M M G BrandsHanka DekkerErica van de MheenMirjam LangeveldCarla E M HollakBarbara Sjouke

Published in: Orphanet journal of rare diseases (2022)

This study underlines the importance of exploring patients' needs and expectations before using limited resources in the development of therapies for patient groups of which a significant subset may not be willing to undergo that specific therapy.

Keyphrases

end stage renal disease
newly diagnosed
chronic kidney disease
gene therapy
prognostic factors
peritoneal dialysis
stem cells
mesenchymal stem cells
case report
patient reported outcomes
bone marrow
patient reported