Publications by authors named "C L Addy"
This is the final of four papers updating standards for the care of people with CF. That this paper "Planning a longer life" was considered necessary, highlights how much CF care has progressed over the past decade. Several factors underpin this progress, notably increased numbers of people with CF with access to CFTR modulator therapy.
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- - This paper is part of a series updating standards of care for cystic fibrosis (CF), emphasizing the importance of recognizing and managing various CF health issues despite advancements like CFTR modulators.
- - It highlights the need for multidisciplinary care to address ongoing extrapulmonary issues such as diabetes, liver disease, and increased cancer risk, which require regular screenings.
- - Mental and emotional support is crucial for individuals with CF and their families, especially during challenging times, and planning for end-of-life care should be approached with sensitivity and compassion.
This is the second in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF. This paper focuses on establishing and maintaining health. The guidance is produced using an evidence-based framework and with wide stakeholder engagement, including people from the CF community.
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- Primary biliary cholangitis (PBC) is a chronic liver disease affecting bile ducts, and the effectiveness of elafibranor, a dual PPAR α and δ agonist, in treating PBC was investigated through a clinical trial.
- In a phase 3, double-blind trial with 161 participants, those treated with elafibranor showed a significant biochemical response (51%) compared to only 4% in the placebo group, indicating substantial improvement in liver function.
- While 15% of elafibranor-treated patients normalized their alkaline phosphatase levels, the results for reducing itching (pruritus) between the elafibranor and placebo groups were not statistically
Article Synopsis
- Advances in cystic fibrosis (CF) diagnosis focus on improvements in newborn bloodspot screening, gene analysis, and understanding CFTR-related disorders.
- These developments highlight the importance of timely and accurate diagnosis for access to variant-specific therapy, which can significantly benefit those with eligible CFTR gene variants.
- The guidance in this paper updates previous standards and emphasizes the growing necessity for proper diagnosis as future trials for variant-specific therapies are anticipated.