Work

Pompe disease in Sweden: A real-world evidence study investigating disease burden, treatment patterns for enzyme replacement therapy and concomitant medications

Year: 2025

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2024.108875

Authors: Sofia Nordin, Natalia M. Stelmaszuk, Alasdair MacCulloch, Ryan Graham, Anne-Berit Ekström, Björn Lindvall, Jonatan Freilich

Institutions: Amicus Therapeutics (United Kingdom), University of Gothenburg, Örebro University Hospital

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Genetics (clinical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4406981966

FWCI:

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