Concepedia

The problems of clinical trials and registries in rare diseases

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

Respiratory Medicine · 2010 · 24 citations · 9 references

DOIFull text

Open access

Concepts

References

9

Pulmonary Alveolar Proteinosis

Samuel H. Rosen, Benjamin Castleman, Averill A. Liebow et al. · New England Journal of Medicine · 1958 · 1.2K citations

, , +17

Idiopathic Pulmonary Alveolar Proteinosis as an Autoimmune Disease with Neutralizing Antibody against Granulocyte/Macrophage Colony-Stimulating Factor

Takayuki Kitamura, Naohiko Tanaka, Junichi Watanabe et al. · The Journal of Experimental Medicine · 1999 · 559 citations · Full text

, , +13

A Randomized Clinical Trial of α1-Antitrypsin Augmentation Therapy

Asger Dirksen, Joop H. Dijkman, Flemming Madsen et al. · American Journal of Respiratory and Critical Care Medicine · 1999 · 499 citations

, , +13

Characteristics of a Large Cohort of Patients with Autoimmune Pulmonary Alveolar Proteinosis in Japan

Yoshikazu Inoue, Bruce C. Trapnell, Ryushi Tazawa et al. · American Journal of Respiratory and Critical Care Medicine · 2008 · 487 citations · Full text

, , +11

Exploring the role of CT densitometry: a randomised study of augmentation therapy in α<sub>1</sub>-antitrypsin deficiency

Asger Dirksen, Eeva Piitulainen, David Parr et al. · European Respiratory Journal · 2009 · 353 citations · Full text

, , +14