Article
Medicine, General & Internal
Bernardo Cabral, Vito Terlizzi, Onofrio Laselva, Carlos Conte Filho, Fabio Mota
Summary: This study assesses the opinions of cystic fibrosis researchers and finds that CFTR modulator therapies and fixing or replacing the CFTR gene are the most likely approaches to successfully treat cystic fibrosis within the next 15 years.
JOURNAL OF CLINICAL MEDICINE
(2022)
Review
Microbiology
Stamatios Gregoriou, Maria Kyriazopoulou, Aikaterini Tsiogka, Dimitrios Rigopoulos
Summary: Onychomycosis, a common nail disease caused by fungi, often results in difficulties in daily life and severe emotional stress. Resistance to antifungals, increasing comorbidities, and polydrug use among the elderly have become challenges in treating onychomycosis. The development of new topical drugs addresses the need for effective agents with prolonged post-treatment disease-free time.
Review
Endocrinology & Metabolism
Amir Moheet, Antoinette Moran
Summary: Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis (CF) and is associated with poor health outcomes. The pathophysiology of CFRD is complex and not fully understood, but factors such as inflammation, alterations in the hormone axis, insulin resistance, and genetic factors may contribute to its development. Further research is needed to better understand the mechanisms underlying CFRD and the impact of diabetes on clinical outcomes in CF.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2022)
Article
Respiratory System
Adele Coriati, Xiayi Ma, Jenna Sykes, Sanja Stanojevic, Rasa Ruseckaite, Lydie Lemonnier, Clemence Dehillotte, Jan Tate, Catherine Ann Byrnes, Scott C. Bell, Pierre Regis Burgel, Anne L. Stephenson
Summary: This study compared the survival rates of cystic fibrosis patients in countries with single-payer healthcare systems, taking into account markers of disease severity. The results showed that France had significantly higher survival rates compared to other countries, despite having a higher proportion of underweight patients.
Article
Allergy
Sara Manti, Giuseppe Fabio Parisi, Maria Papale, Gian Luigi Marseglia, Amelia Licari, Salvatore Leonardi
Summary: Type 2 inflammation is recognized as a critical contributor to cystic fibrosis, and understanding its pathways can help identify targets for treating inflammation and fibrosis in patients with CF.
PEDIATRIC ALLERGY AND IMMUNOLOGY
(2022)
Review
Dermatology
Ping-Chen Hou, Han-Tang Wang, Stasha Abhee, Wei-Ting Tu, John A. McGrath, Chao-Kai Hsu
Summary: Epidermolysis bullosa is a rare inherited blistering skin disorder characterized by skin fragility since birth, with treatment mainly focusing on wound care and patient symptoms. Recent therapeutic advances such as gene therapies, cell-based therapies, and drug repurposing approaches offer new hope for better patient care and improved clinical management.
AMERICAN JOURNAL OF CLINICAL DERMATOLOGY
(2021)
Article
Respiratory System
Emily Granger, Gwyneth Davies, Ruth H. Keogh
Summary: This study investigates whether adding hypertonic saline to patients with cystic fibrosis who are already using dornase alfa has additional benefits for lung function or use of intravenous antibiotics. The findings indicate that adding hypertonic saline does not significantly improve lung function or decrease the use of intravenous antibiotics in cystic fibrosis patients.
Review
Pediatrics
Claire Edmondson, Christopher William Course, Iolo Doull
Summary: Cystic fibrosis, a genetic disorder, has been significantly improved by the new CFTR modulators, leading to increased life expectancy for patients. However, the high cost is the main barrier to their wider introduction.
ARCHIVES OF DISEASE IN CHILDHOOD
(2021)
Review
Health Care Sciences & Services
Christos Damaskos, Nikolaos Garmpis, Anna Garmpi, Konstantinos Nikolettos, Panagiotis Sarantis, Vasiliki E. Georgakopoulou, Afroditi Nonni, Dimitrios Schizas, Efstathios A. Antoniou, Michalis Karamouzis, Nikos Nikolettos, Konstantinos Kontzoglou, Alexandros Patsouras, Errika Voutyritsa, Athanasios Syllaios, Evangelos Koustas, Nikolaos Trakas, Dimitrios Dimitroulis
Summary: Triple-negative breast cancer is an aggressive subtype of breast cancer that does not respond well to hormone therapy but often shows good response to chemotherapy. It is important to investigate new beneficial targeted therapies in order to achieve enhanced outcomes for patients.
JOURNAL OF PERSONALIZED MEDICINE
(2021)
Review
Biochemistry & Molecular Biology
Christelle Bergeron, Andre M. Cantin
Summary: Rare diseases affect 400 million individuals globally, and cystic fibrosis, a genetic disease, has seen advancements in potential therapies that aim to restore CFTR function, showing promise in improving the lives of patients.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Nutrition & Dietetics
Carmen Mangas-Sanchez, Maria Garriga-Garcia, Maria Juliana Serrano-Nieto, Ruth Garcia-Romero, Marina alvarez-Beltran, Elena Crehua-Gaudiza, Rosana Munoz-Codoceo, Lucrecia Suarez-Cortina, Saioa Vicente-Santamaria, Cecilia Martinez-Costa, Juan Jose Diaz-Martin, Carlos Bousono-Garcia, David Gonzalez-Jimenez
Summary: In patients with CF, higher doses of vitamin D were associated with a decreased risk of deficiency and higher serum calcidiol levels. However, nearly one-third of CF patients still do not reach sufficient levels of vitamin D despite the new guidelines.
Review
Pediatrics
Marlou C. Bierlaagh, Danya Muilwijk, Jeffrey M. Beekman, Cornelis K. van der Ent
Summary: Cystic fibrosis is a genetic disease caused by a defect in the CFTR gene, with pulmonary manifestations being the most significant threat. However, the introduction of CFTR modulators since 2012 has significantly improved life expectancy and quality of life for cystic fibrosis patients.
EUROPEAN JOURNAL OF PEDIATRICS
(2021)
Review
Biochemistry & Molecular Biology
Luigino Calzetta, Marina Aiello, Annalisa Frizzelli, Elena Pistocchini, Beatrice Ludovica Ritondo, Paola Rogliani, Alfredo Chetta
Summary: Inhaled corticosteroids (ICS) are the main treatment for asthma, but there is a need for innovative medications that selectively target components of inflammation. A review of recent randomized controlled trials identified 16 classes of novel therapeutic options, with lung function being the main assessed outcome. Promising disease-modifying agents, particularly biologics, are being studied, but further research is needed to confirm positive results from Phase I and II trials.
Review
Endocrinology & Metabolism
Sarah S. Malik, Diksha Padmanabhan, Rebecca L. Hull-Meichle
Summary: Cystic fibrosis related diabetes is a common complication in patients with cystic fibrosis, but the mechanisms behind its occurrence are not well understood. Cystic fibrosis affects the pancreatic/islet microenvironment, leading to the loss of beta cell function and pathological changes. Further research can help develop better treatment methods.
FRONTIERS IN ENDOCRINOLOGY
(2023)
Review
Medicine, General & Internal
Sasan Sakiani, Theo Heller, Christopher Koh
Summary: Treatment options for portal hypertension are limited and mainly focus on reducing hyperdynamic circulation and treating hypervolemia. Newer and more effective treatment options are needed to improve survival and quality of life.
FRONTIERS IN MEDICINE
(2022)