The problems of clinical trials and registries in rare diseases - 20/08/11
, Ilaria Campo, Roberta Scabini, Michele Zorzetto, Zamir Kadija, Francesca Mariani, Ilaria Ferrarotti
Corresponding author. Tel.: +39 0382 423131; fax: +39 0382 42267.Summary |
Clinical 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.
Le texte complet de cet article est disponible en PDF.Keywords : Rare diseases, Alpha1-antitrypsin deficiency, Pulmonary alveolar proteinosis, GM-CSF, Alpha1-antitrypsin, Aerosol delivery of drugs
Plan
Vol 104 - N° S1
P. S42-S44 - juillet 2010 Retour au numéro
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