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Open Access 01-12-2014 | Oral presentation

The experience of a charity in translating the results of basic research to therapies for patients

Author: Lucia Monaco

Published in: Orphanet Journal of Rare Diseases | Special Issue 1/2014

Excerpt

Rare diseases represent a relevant societal challenge that calls different players into action, with the aim of providing a diagnosis to patients, understanding the disease pathophysiology and, most importantly, developing treatments and therapies to effectively improve life quality and expectancy. …

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Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, Callegaro L, Benati C, Rizzardi P, Pellin D, Di Serio C, Schmidt M, Von Kalle C, Gardner J, Mehta N, Neduva V, Dow DJ, Galy A, Miniero R, Finocchi A, Metin A, Banerjee PP, Orange JS, Galimberti S, Valsecchi MG, Biffi A, Montini E, Villa A, Ciceri F, Roncarolo MG, Naldini L: Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science. 2013, 341: 1233151-10.1126/science.1233151.PubMedCentralCrossRefPubMed

Title: The experience of a charity in translating the results of basic research to therapies for patients
Author: Lucia Monaco
Publication date: 01-12-2014
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue Special Issue 1/2014
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/1750-1172-9-S1-O20

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