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Personalised medicine for cystic fibrosis: treating the basic defect

J. Stuart Elborn
European Respiratory Review 2013 22: 3-5; DOI: 10.1183/09059180.00008112
J. Stuart Elborn
Centre for Infection and Immunity, School of Medicine, Dentistry and Biomedical Sciences, Queens University, Belfast, UK
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THE RIGHT TREATMENT, FOR THE RIGHT PATIENT, AT THE RIGHT TIME

The concept of personalised or stratified therapies is not a new one. For centuries, physicians have observed that the manifestation of a disease and its response to intervention can vary according to many factors including age, sex, ethnicity, diet and the type of administered drug [1]. Hippocrates, for example, observed over 2,000 years ago that patients can respond very differently to various medications [2].

It wasn’t until 1998, however, that the term “personalised therapy” was first used [3]. This can be defined as a therapy prescribed using molecular profiling technologies to tailor the right therapeutic strategy for the right person at the right time. The therapy is typically accompanied by a “companion test” or clinical biomarker to identify responders whilst assessing therapeutic response.

Personalised medicine is often used synonymously with the expression “stratified medicine”. There are, however, subtle differences between both of these terms as illustrated by the patient therapeutic continuum described by Trusheim et al. (fig. 1) [4].

Figure 1.

The spectrum of therapeutic care: from empirical medicine to personalised medicine. PPI: proton pump inhibitor; SSRI: serotonin reuptake inhibitor; NSAID: non-steroidal anti-inflammatory drug.

Most of today's medical practice is empirical, with drug development targeted at treating large populations of patients; an example is non-steroidal anti-inflammatory drugs, which have high efficacy in almost the …

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Personalised medicine for cystic fibrosis: treating the basic defect
J. Stuart Elborn
European Respiratory Review Mar 2013, 22 (127) 3-5; DOI: 10.1183/09059180.00008112

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Personalised medicine for cystic fibrosis: treating the basic defect
J. Stuart Elborn
European Respiratory Review Mar 2013, 22 (127) 3-5; DOI: 10.1183/09059180.00008112
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  • Article
    • THE RIGHT TREATMENT, FOR THE RIGHT PATIENT, AT THE RIGHT TIME
    • PERSONALISED MEDICINE AND RESPIRATORY DISEASE
    • CYSTIC FIBROSIS AS A PARADIGM FOR PROSPECTIVE PERSONALISED DRUG DEVELOPMENT
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  • Interstitial and orphan lung disease
  • Genetics
  • CF and non-CF bronchiectasis
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  • Clinical and prognostic value of ventilatory efficiency in cardiorespiratory disorders
  • Guidance production before evidence generation for critical issues: COVID-19
  • Modulator treatment for people with cystic fibrosis
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