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Lifetime impact of achondroplasia study in Europe (LIAISE): findings from a multinational observational study

15 Mar 2023

Abstract excerpt

BACKGROUND: Achondroplasia, caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene, is the most common skeletal dysplasia. The Lifetime Impact of Achondroplasia Study in Europe (LIAISE; NCT03449368) aimed to quantify the burden of achondroplasia among individuals across a broad range of ages, including adults. METHODS: Demographic, clinical and healthcare resource use data were collected...

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PMID: 36922864
DOI: 10.1186/s13023-023-02652-2

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