That includes an international group of researchers; leading drug developers that includes multinational pharmaceutical companies and smaller biotechnology firms; clinical registries from the leading research institutions in Duchenne muscular dystrophy; and pioneering advocacy organizations that have led the way in the fight against Duchenne.
Clinical Centres and Clinical Networks
De-identified patient data shared with cTAP has been collected by major neuromuscular paediatric care centres, as well as by networks of clinical centres, primarily from Europe and North America; it includes natural history, real world and clinical trial data.
We are grateful to these clinical centres, clinical networks and their staff for curating patient data; to patient organizations for their financial support, and to drug companies for the clinical trial data they shared with cTAP.
Finally, we thank all patients and their families for consenting their data for research.
Fondazione Telethon, Italy
iMDEX and AFM-Téléthon, France
Northstar UK and Muscular dystrophy UK
Children’s Hospital of Philadelphia
Cincinnati Children’s Hospital Medical Centre, USA
Pediatrics and Child Neurology, University Hospitals Leuven, Belgium
Biomarin and CureDuchenne
Joint Steering Committee
Astellas Pharma: Makoto Kashiwa, Ph.D. Medicine, D.M.V., Executive Director
Catabasis: Joanne Donovan, M.D., Ph.D., Chief Medical Officer
Charley’s Fund: Laura Dalle Pazze, A.B., President and Chief Operating Officer
CureDuchenne: Debra Miller, B.A., Founder and CEO
FibroGen, Inc.: Elias Kouchakji, M.D., Sr. Vice President, Clinical Development, Drug Safety and Pharmacovigilance
Genentech: Karl Yen M.D., M.M.Sc, Associate Group Medical Director
Italfarmaco SpA: Paolo Bettica, M.D., Vice President, Research and Development
Parent Project Muscular Dystrophy: Abby Bronson, M.B.A., Senior Vice President, Research Strategy
Pfizer, Inc.: Michael Binks, M.D., Vice President for Rare Disease Clinical Research
PTC Therapeutics: Francesco Bibbiani, M.D., Vice President, Clinical Development
Sarepta Therapeutics: Ashish Duggar Ph.D., M.B.A, Vice President, Global Medical Affairs
Shire plc: Csaba Siffel, M.D., Ph.D., P.M.P., Director of Pharmacoepidemiology
Solid Biosciences: Jorge Quiroz, M.D., Chief Medical Officer
Wave Life Sciences: Stephen Lake, Ph.D., Vice President, Biometrics
Collaboration Management Team
Susan J. Ward, Ph.D., Founder and Executive Director, cTAP
James Signorovitch, Ph.D., Managing Principal, Analysis Group
Gautam Sajeev, Ph.D., Manager, Analysis Group
Seden Grippon, Ph.D., Project Manager, cTAP; Consultant, Grippon Consultancy
Katherine Beaverson, Communications Lead, cTAP • Senior Director and Patient Advocacy Lead, Rare Disease Research Unit, Pfizer, Inc