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Biomarkers and surrogate endpoints for drug development in propionic acidemia

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Year: 2026
Type: article
Source: Molecular Genetics and Metabolism
DOI: 10.1016/j.ymgme.2026.110202
Authors: Agnieszka Jurecka, Sabine Scholl-Bürgi, Barbara Burton, Roberto Zori, Nicola Longo, Jerry Vockley, Carlo Dionisi-Vici
Institutions: Harvard University, Innsbruck Medical University, Universität Innsbruck, Northwestern University, Lurie Children's Hospital, University of Florida, University of California, Los Angeles, University of Pittsburgh, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Bambino Gesù Children's Hospital
Language: English

Topic: Metabolism and Genetic Disorders
Subfield: Clinical Biochemistry
Field: Biochemistry, Genetics and Molecular Biology
Domain: Life Sciences
SDG: Good health and well-being

Open Access status: closed
OpenAlex ID: W7167332324
FWCI:
Cites: 60
Cited by:
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