Cystic Fibrosis: Clinical Manifestations and Treatment

Dear Colleagues,

Over the last few decades, significant progress has been made in medical innovations related to cystic fibrosis (CF), a chronic and life-threatening genetic disorder that affects both the digestive and respiratory systems. Although CF is a monogenic disease, people with the same pathogenic mutation may have a variable phenotype due to the intervention of modifier genes or environmental factors. The research on modifier genes opens up new perspectives, both in terms of the diagnosis and prognosis of the disease as well as therapeutic interventions.

Following the introduction of screening programs, the incidence of the disease has been more accurately documented, enabling healthcare providers to initiate treatment and management strategies promptly, ultimately improving the quality of life for individuals with CF. Moreover, new treatments such as CFTR (cystic fibrosis transmembrane conductance regulator) modulators have revolutionized the treatment of CF by targeting the underlying cause of the disease.

However, treating a child with CF still presents a myriad of challenges due to the complex and progressive nature of the disease. Although the majority of the CF population is benefiting from the CFTR modulators, there are still people with CF without a treatment program targeting their specific genetic mutations. Since monitoring the disease is crucial for achieving positive outcomes, it is essential to properly assess and manage new techniques such as liver elastography and continuous glycemia monitoring. In this context, personalized medicine approaches hold promise for further advancements in the management of CF in children, ultimately leading to better outcomes and improved life expectancy for individuals with this condition.

We invite researchers, clinicians, and other experts in the field to contribute their insights and expertise to this Special Issue. We are looking for reviews and original articles reflecting the latest progress in the field of clinical and molecular diagnostics, as well as novel therapeutic approaches. Topics include, but are not limited to, the following:

• Clinical manifestations and complications of cystic fibrosis in different organ systems;
• Novel diagnostic tools and biomarkers for the early detection of cystic fibrosis;
• Pharmacological and non-pharmacological treatment strategies for cystic fibrosis;
• Nutritional care in children with cystic fibrosis;
• Emerging therapies and personalized medicine approaches for cystic fibrosis management.

We hope that by sharing knowledge and collaborating on new ideas, we can work together to enhance our understanding of CF in children and ultimately improve the care and outcomes for these young patients.

Prof. Dr. Laura Trandafir
Dr. Lǎcrǎmioara Ionela Butnariu
Guest Editors

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• cystic fibrosis
• modifier genes
• CFTR modulator
• CF newborn screening
• CF nutrition
• CF complications