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pubmed-article:20413285pubmed:abstractTextClinical trials to evaluate patients affected by rare diseases are often hampered by the difficulty of recruiting a critical sample size. Registries for rare conditions are thus extremely powerful tools for overcoming recruitment problems. Here we present and discuss the international experience with alpha1-antitrypsin deficiency achieved by the Alpha One International Registry, and national experience obtained with a large series of patients with pulmonary alveolar proteinosis.lld:pubmed
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pubmed-article:20413285pubmed:authorpubmed-author:ZorzettoMiche...lld:pubmed
pubmed-article:20413285pubmed:authorpubmed-author:FerrarottiIla...lld:pubmed
pubmed-article:20413285pubmed:authorpubmed-author:LuisettiMauri...lld:pubmed
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pubmed-article:20413285pubmed:authorpubmed-author:MarianiFrance...lld:pubmed
pubmed-article:20413285pubmed:authorpubmed-author:KadijaZamirZlld:pubmed
pubmed-article:20413285pubmed:copyrightInfoCopyright (c) 2010 Elsevier Ltd. All rights reserved.lld:pubmed
pubmed-article:20413285pubmed:issnTypeElectroniclld:pubmed
pubmed-article:20413285pubmed:volume104 Suppl 1lld:pubmed
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pubmed-article:20413285pubmed:year2010lld:pubmed
pubmed-article:20413285pubmed:articleTitleThe problems of clinical trials and registries in rare diseases.lld:pubmed
pubmed-article:20413285pubmed:affiliationClinica di Malattie dell'Apparato Respiratorio, Fondazione IRCCS Policlinico San Matteo, Università di Pavia, Via Golgi 19, 27100 Pavia, Italy. m.luisetti@smatteo.pv.itlld:pubmed
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