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FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis
- Sven F. Garbade,
- Matthias Zielonka,
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Konstantin Mechler,
Roles Formal analysis, Writing – review & editing
Affiliation Department of Child and Adolescent Psychiatry and Psychotherapy & Department of Addictive Behavior and Addiction Medicine, Medical Faculty Mannheim, Central Institute of Mental Health, University of Heidelberg, Mannheim, Germany
⨯ - Stefan Kölker,
- Georg F. Hoffmann,
- Christian Staufner,
- Eugen Mengel,
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Markus Ries
Roles Conceptualization, Formal analysis, Investigation, Methodology, Supervision, Validation, Writing – original draft
* E-mail: markus.ries@uni-heidelberg.de
Affiliations Division of Pediatric Neurology and Metabolic Medicine, Center for Pediatric and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany, Center for Rare Diseases, University Hospital Heidelberg, Heidelberg, Germany, Center for Virtual Patients, Medical Faculty, University of Heidelberg, Heidelberg, Germany
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http://orcid.org/0000-0002-5322-7140
FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis
- Sven F. Garbade,
- Matthias Zielonka,
- Konstantin Mechler,
- Stefan Kölker,
- Georg F. Hoffmann,
- Christian Staufner,
- Eugen Mengel,
- Markus Ries
- Published: April 8, 2020
- https://doi.org/10.1371/journal.pone.0230898