Article

Patient and Family Experiences of Lysosomal Storage Diseases: A Qualitative Interview Study

2022-09-09

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold> Patients and families affected by rare genetic lysosomal storage diseases (LSDs) suffer from a variety of challenges related to disease management, including issues navigating healthcare and social support services, access to orphan drugs, and intensive treatment regimens. All of these challenges significantly impact people’s quality of life, yet they remain obsc...

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Literature Corpus work: dfbd94f0-25c5-5e04-b576-ee44d26e1cf1
DOI: 10.21203/rs.3.rs-2031190/v1

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