Treatment Goals for Achondroplasia: A Qualitative Study with Parents and Adults.

ABSTRACT:

Introduction

Achondroplasia is characterized by disproportionate short stature accompanied by other changes to the musculoskeletal system. Individuals with this condition typically experience a variety of medical complications. As pharmacologic treatments continue to be developed for the treatment of achondroplasia, it is important to understand treatment goals among those affected by achondroplasia and the factors that shape their goals.

Methods

This qualitative study is based on semi-structured interviews with 19 parents of children with achondroplasia and five adults with achondroplasia in the USA. We employed thematic analysis using an iterative process to identify themes across the interviews.

Results

Participants had two goals for pharmacologic treatment of acho

SUBMITTER: McGraw SA 

PROVIDER: S-EPMC9239927 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature