Rare diseases and effective treatments: are we delivering?

Affiliations

• ¹ Istituto Toscano Tumori, Florence, Italy.
• ² Department of Endocrinology and Metabolism, Academic Medical Center, Amsterdam, the Netherlands.
• ³ University Department of Medicine, University of Cambridge, Cambridge, UK.
• ⁴ IRCCS Istituto di Ricerche Farmacologiche Mario Negri, 24126 Bergamo, Italy; Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.
• ⁵ Division of Nephrology, The Hospital for Sick Children, Toronto, Canada.
• ⁶ European Platform for Patients' Organizations Science and Industry, Brussels, Belgium.
• ⁷ Amyloidosis Research and Treatment Center, Department of Molecular Medicine, University of Pavia, Italy.
• ⁸ Pediatric Nephrology Division, Heidelberg University Center for Pediatrics and Adolescent Medicine, Heidelberg, Germany.
• ⁹ KU Leuven Department of Pharmaceutical and Pharmacological Sciences, Leuven, Belgium.
• ¹⁰ AIFA Italian Medicine Agency, Rome, Italy.
• ¹¹ IRCCS Istituto di Ricerche Farmacologiche Mario Negri, 24126 Bergamo, Italy.
• ¹² IRCCS Istituto di Ricerche Farmacologiche Mario Negri, 24126 Bergamo, Italy. Electronic address: giuseppe.remuzzi@marionegri.it.