Acknowledgements
Daniela Dragone, Adele Valletta
BREDA
The authors acknowledge Prof. Monika Engelhardt (University of Freiburg, Germany), Prof. Martin Gramatzi (University of Kiel, Germany), and Prof. Klaus Podar (University of Krems, Austria) for being valuable members of our Scientific Advisory Board; Prof. Paola Ernesta Neri (University of Calgary, Canada), Prof. Emilio Russo (Magna Graecia University, Italy) and Dr. Pierpaolo Correale (GOM RC, Italy) for being valuable members of the SRC; Dr. Niels Montano Frandsen (Exiqon/Qiagen, Vedbaek Copenhagen, Denmark) as an expert external consultant on LNA and ASO chemistry; Dr. Massimo Breda (Aptuit/Evotec, Verona, Italy) for GLP/GCP Compliance in PK data production; Dr. Massimiliano Fonsi (Citoxlab/ Charles River Laboratories Evreux, France) as a supervisor on design and modelling of PK data; Dr. Brian Sproat (Managing Director at Chemconsilium GCV, Belgium) for the drug substance dossier; Dr. Loredana Cecchetelli (Director R&D at IBI-Istituto Biochimico Italiano Giovanni Lorenzini Spa, Aprilia-Rome) for GMP production support, fill&finish process and dossier; Prof. Marco Rossi for critical suggestions on the clinical protocol; Dr. Francesco Grillone for selecting the first patient to be enrolled in the study; all the nursing staff, in particular Dr. Rosina Pane, Dr. Daniela Dragone, Dr. Vincenza Aversa, Dr. Emanuele Montauro, Dr. Simona Tarzia, Dr. Luana Iannone, Dr. Franca Malvaggio, Dr. Francesca Ceravolo, Dr. Adele Valletta, and Dr. Marianna Arcidiacono, for their dedication and precious work on the project within the Phase I Unit; Dr. Ivana Criniti for study support and secretarial assistance; and Magnus Ringbom (Director, Medical Writing) and Dr. Christopher Furniss (Medical Writer) from LINK Medical Research AB, Sweden, who assisted in the preparation of the first draft of the manuscript. Our greatest thanks go to the patients and their families who participated in this study.