Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.

ABSTRACT:

Background

Lysosomal storage diseases (LSDs) are inherited metabolic disorders that may lead to severe multi-organ disease. Current ERTs are limited by anti-drug antibodies, the blood-brain barrier, and early disease onset and progression before ERT is started. We have opened a phase I clinical trial of enzyme replacement therapy (ERT) for fetuses with LSDs (NCT04532047). We evaluated the attitudes of parents and patients with LSDs towards fetal clinical trials and therapies.

Methods

A multidisciplinary team designed a survey which was distributed by five international patient advocacy groups. We collected patients' demographic, diagnostic, and treatment information. Associations between respondent characteristics and attitudes towards fetal therapies/trials were analyzed us

SUBMITTER: Schwab ME 

PROVIDER: S-EPMC8800365 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature