About the Authors

Richard W. D. Welford

richard.welford@actelion.com

Affiliation Actelion Pharmaceuticals Ltd, Allschwil, Switzerland

Marco Garzotti

Affiliation Actelion Pharmaceuticals Ltd, Allschwil, Switzerland

Charles Marques Lourenço

Affiliation Hospital das Clínicas de Ribeirão Preto, São Paulo, Brazil

Eugen Mengel

Affiliation Department of Lysosomal Storage Disorder, Villa Metabolica, Center for Paediatric and Adolescent Medicine, University Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

Thorsten Marquardt

Affiliation Klinik für Kinder- und Jugendmedizin, Münster, Germany

Janine Reunert

Affiliation Klinik für Kinder- und Jugendmedizin, Münster, Germany

Yasmina Amraoui

Affiliation Department of Lysosomal Storage Disorder, Villa Metabolica, Center for Paediatric and Adolescent Medicine, University Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

Stefan A. Kolb

Affiliation Actelion Pharmaceuticals Ltd, Allschwil, Switzerland

Olivier Morand

Affiliation Actelion Pharmaceuticals Ltd, Allschwil, Switzerland

Peter Groenen

Affiliation Actelion Pharmaceuticals Ltd, Allschwil, Switzerland

Competing Interests

RW, MG, SK, OM and PG are employees of Actelion Pharmaceuticals Ltd; CML, EM, YA, JR and TM have received travel reimbursements, consulting fees or honoraria from Actelion Pharmaceuticals Ltd, Allschwil, Switzerland. The competing interests do not alter the authors' adherence to PLOS ONE policies on sharing data and materials.

Author Contributions

Conceived and designed the experiments: RW MG CML EM TM JR YA SK OM PG. Performed the experiments: MG. Analyzed the data: RW MG OM PG. Contributed reagents/materials/analysis tools: CML EM TM JR YA. Wrote the paper: RW MG CML EM TM SK OM PG.