TY - JOUR T1 - Rare pulmonary diseases and orphan drugs: where do we stand and where are we going to? JF - European Respiratory Review JO - EUROPEAN RESPIRATORY REVIEW SP - 375 LP - 377 DO - 10.1183/16000617.0051-2015 VL - 24 IS - 137 AU - Sergio Harari AU - Marc Humbert AU - Francesco Blasi AU - Harold R. Collard AU - Marco Matucci-Cerinic AU - Gerald Simonneau AU - Carlo Vancheri AU - Athol U. Wells AU - Vincent Cottin Y1 - 2015/09/01 UR - http://err.ersjournals.com/content/24/137/375.abstract N2 - Rare diseases are a major problem for the patients who suffer from them, for their families and for selected specialists involved in their management. In addition, they cause economic, social and public health problems that should be acknowledged and discussed by the medical and scientific community. Because of the rarity of these illnesses, the experience of most physicians is usually limited to a few cases. Nevertheless, the essential steps in the diagnosis and management of a patient who is suspected to have, for example, an interstitial lung disease or pulmonary hypertension, should be common knowledge, as these diseases are uncommon, but not, in fact, exceedingly rare [1–3]. Furthermore, rare presentations of frequent lung diseases (e.g. rare thoracic cancers, severe asthma, and emphysema with α1-antitrypsin deficiency) and the most typical rare pulmonary diseases can share similar aspects, both in research and in their clinical management [4].Updates on rare pulmonary diseases from the 6th International Meeting on Pulmonary Rare Diseases and Orphan Drugs http://ow.ly/PYUCC ER -