Living with Pompe disease in the UK: Characterizing the patient journey and burden on physical, emotional and social quality of life
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DOI: 10.1016/j.ymgme.2021.11.145
Abstract: Pompe disease is a rare, progressive, multisystemic disease with heterogenous presentation. We evaluated the burden, unmet needs and evolving management landscape for people living with late-onset Pompe disease (LOPD) based on their own experiences. The… read more here.
Keywords: quality life; living lopd; disease; journey ... See more keywords