Further reading

General

  • Ayme S, Kole A, Groft S. Empowerment of patients: lessons from the rare diseases. Lancet 2008; 371: 2048–2051.
  • Buckley BM. Clinical trials of orphan medicines. Lancet 2008; 371: 2051–2055.
  • Clarke JTR, Giugliani R, Sunder-Plassmann G, et al. Impact of measures to enhance the value of observational surveys in rare diseases: the Fabry Outcome Survey (FOS). Value Health 2011; 14: 862–866.
  • Cordier JF. Rare and orphan lung diseases. Breathe 2005; 2: 147–154.
  • Cordier JF, ed. Orphan Lung Diseases. Eur Respir Monogr 2011; 54.
  • Desser AS, Gyrd-Hansen D, Olsen JA, et al. Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67. BMJ 2010; 341: c4715.
  • Garg NJ. Global health: neglected diseases and access to medicines. Infect Dis Clin NorthAm 2011; 25: 639–651.
  • Hotez PJ, Fenwick A, Molyneux DH. Rescuing the bottom billion through control of neglected tropical diseases. Lancet 2009; 373: 1570–1575.
  • Ingelfinger JR, Drazen JM. Patient organizations and research on rare diseases. N Engl JMed 2011; 364: 1670–1671.
  • Llera VA, Roldan EJA. Postmarketing trials for rare diseases. Science 2012; 337: 154.
  • Luisetti M, Balfour-Lynn IM, Johnson SR, et al. Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe. Respir Med 2012; 106: 759–768.
  • Rath A, Olry A, Dhombres F, et al. Representation of rare diseases in health information systems: the orphanet approach to serve a wide range of end users. Hum Mutat 2012; 33:803–805.
  • Simoens S, Cassiman D, Dooms M, et al. Orphan drugs for rare diseases. Is it time to revisit their special market access status? Drugs 2012; 72: 1437–1443.

Specific diseases

  • Hoeper MM, Humbert M, eds. Pulmonary Hypertension. Eur Respir Monogr 2012; 57.
  • Johnson SR, Cordier JF, Lazor R, et al. European Respiratory Society guidelines for the diagnosis and management of lymphangioleiomyomatosis. Eur Respir J 2010; 35: 14–26.
  • Lazor R, Etienne-Mastroianni B, Khouatra C, et al. Progressive diffuse pulmonary Langerhans cell histiocytosis improved by cladribine chemotherapy. Thorax 2009; 64:274–275.
  • Lorillon G, Bergeron A, Detoumignies L, et al. Cladribine is effective against cystic pulmonary Langerhans cell histiocytosis. Am J Respir Crit Care Med 2012; 186: 930–932.
  • Nelson D, Specks U. Granulomatosis with polyangiitis. In: Cordier JF, ed. Orphan Lung Diseases. Eur Respir Monogr 2011; 54; pp. 1–14.
  • Lazor R. Alveolar haemorrhage syndromes. In: Cordier JF, ed. Orphan Lung Diseases. EurRespir Monogr 2011; 54; pp. 15–31.
  • Cottin V. Idiopathic eosinophilic pneumonias. In: Cordier JF, ed. Orphan Lung Diseases. EurRespir Monogr 2011; 54; pp. 118–139.
  • Guillevin L, Dunogue B, Pagnoux C. Churg–Strauss syndrome. In: Cordier JF, ed. Orphan Lung Diseases. Eur Respir Monogr 2011; 54; pp. 140–151.
  • Bonella F, Theegarten D, Guzman J, et al. Alveolar lipoproteinosis syndromes. In: Cordier JF,ed. Orphan Lung Diseases. Eur Respir Monogr 2011; 54; pp. 171–186.
  • Ryu JH, Maldonado F, Tomassetti S. Idiopathic tracheopathies. In: Cordier JF, ed. Orphan Lung Diseases. Eur Respir Monogr 2011; 54; pp. 187–200.
  • Lucas JSA, Walker WT, Kuehni CE, et al. Primary ciliary dyskinesia. In: Cordier JF, ed.Orphan Lung Diseases. Eur Respir Monogr 2011; 54; pp. 201–217.
  • Bobbio A, Trisolini R, Damotte D, et al. Thoracic endometriosis and catamenial pneumothorax. In: Cordier JF, ed. Orphan Lung Diseases. Eur Respir Monogr 2011; 54; pp. 265–281.

See the entire Rare and orphan lung diseases Chapter