Rare diseases and effective treatments: are we delivering?
Istituto Toscano Tumori, Florence, Italy.
Lucio
L
Luzzatto
Department of Endocrinology and Metabolism, Academic Medical Center, Amsterdam, the Netherlands.
Carla E M
CE
Hollak
University Department of Medicine, University of Cambridge, Cambridge, UK.
Timothy M
TM
Cox
IRCCS Istituto di Ricerche Farmacologiche Mario Negri, 24126 Bergamo, Italy; Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.
Arrigo
A
Schieppati
Division of Nephrology, The Hospital for Sick Children, Toronto, Canada.
Christoph
C
Licht
European Platform for Patients' Organizations Science and Industry, Brussels, Belgium.
Helena
H
Kääriäinen
Amyloidosis Research and Treatment Center, Department of Molecular Medicine, University of Pavia, Italy.
Giampaolo
G
Merlini
Pediatric Nephrology Division, Heidelberg University Center for Pediatrics and Adolescent Medicine, Heidelberg, Germany.
Franz
F
Schaefer
KU Leuven Department of Pharmaceutical and Pharmacological Sciences, Leuven, Belgium.
Steven
S
Simoens
AIFA Italian Medicine Agency, Rome, Italy.
Luca
L
Pani
IRCCS Istituto di Ricerche Farmacologiche Mario Negri, 24126 Bergamo, Italy.
Silvio
S
Garattini
IRCCS Istituto di Ricerche Farmacologiche Mario Negri, 24126 Bergamo, Italy. Electronic address: giuseppe.remuzzi@marionegri.it.
Giuseppe
G
Remuzzi
D003695:Delivery of Health Care / Q000592:standards; D016527:Drug Costs; D004345:Drug Industry / Q000191:economics; D006801:Humans; D055118:Medication Adherence; D009042:Motivation; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000191:economics