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Lancet. 2015 Feb 28;385(9970):750-2. doi: 10.1016/S0140-6736(15)60297-5.

Rare diseases and effective treatments: are we delivering?

Author information

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.
PMID:
25752159
DOI:
10.1016/S0140-6736(15)60297-5
[Indexed for MEDLINE]

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