The problems of clinical trials and registries in rare diseases

Maurizio Luisetti, Ilaria Campo, Roberta Scabini, Michele Zorzetto, Zamir Kadija, Francesca Mariani, Ilaria Ferrarotti

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish
JournalRespiratory Medicine
Volume104
Issue numberSUPPL. 1
DOIs
Publication statusPublished - Jul 2010

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Keywords

  • Aerosol delivery of drugs
  • Alpha1-antitrypsin
  • Alpha1-antitrypsin deficiency
  • GM-CSF
  • Pulmonary alveolar proteinosis
  • Rare diseases

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

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