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Effective treatment in the form of cystic fibrosis transmembrane regulator (CFTR) modulator therapy targeting the most common gene mutations is now available for over 90% of people with cystic fibrosis over the age of 2 years. This treatment results in improvement of lung function and nutritional status. However, it is not known whether these effects will be sustained or have a wider influence on the multiple organs affected by cystic fibrosis if introduced early in life. Hence, many Australian people with cystic fibrosis will continue to live with the possibility of organ failure, albeit fewer than in previous generations. There is agreement that single organ transplantation in a patient with organ failure is medically appropriate and ethically justifiable. 

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Authors: Mark R Oliver, John Massie, Miranda Paraskeva, Avik Majumdar, Lynn H Gillam and Dominic JC Wilkinson

Article Type: Ethics and Law