Quantitative retrospective natural history modeling for orphan drug development

Author:

Garbade Sven F.12,Zielonka Matthias12,Komatsuzaki Shoko3,Kölker Stefan12,Hoffmann Georg F.12,Hinderhofer Katrin4,Mountford William K.5,Mengel Eugen6,Sláma Tomáš7,Mechler Konstantin8,Ries Markus129ORCID

Affiliation:

1. Division of Pediatric Neurology and Metabolic Medicine Center for Pediatric and Adolescent Medicine, University Hospital Heidelberg Heidelberg Germany

2. Center for Rare Diseases University Hospital Heidelberg Heidelberg Germany

3. Institute of Human Genetics, University Hospital Jena, Friedrich‐Schiller‐University Jena Jena Germany

4. Institute of Human Genetics, Heidelberg University Heidelberg Germany

5. Department of Clinical Research School of Nursing, College of Health and Human Services, University of North Carolina at Wilmington Wilmington North Carolina USA

6. SphinCS Clinical Science for LSD Hochheim Germany

7. Department of Pediatrics Oberwallis Hospital Visp Switzerland

8. Pediatric Psychopharmacology, Department of Child and Adolescent Psychiatry and Psychotherapy Central Institute of Mental Health, Medical Faculty Mannheim, University of Heidelberg Mannheim Germany

9. Center for Virtual Patients, Medical Faculty University of Heidelberg Heidelberg Germany

Publisher

Wiley

Subject

Genetics(clinical),Genetics

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