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Seminars in Respiratory and Critical Care Medicine
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#1Yasmine Nivoix (UDS: University of Strasbourg)H-Index: 13
#2Marie-Pierre Ledoux (UDS: University of Strasbourg)H-Index: 4
Last.Raoul Herbrecht (UDS: University of Strasbourg)H-Index: 66
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#1Marwan M. Azar (Yale University)H-Index: 13
#2James L. Loyd (Vandy: Vanderbilt University)
Last.Chadi A. Hage (IU: Indiana University)H-Index: 29
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#1Samantha Jacobs (ISMMS: Icahn School of Medicine at Mount Sinai)H-Index: 1
#2Nancy L. Wengenack (Mayo Clinic)H-Index: 33
Last.Thomas J. Walsh (Cornell University)H-Index: 133
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#1Flá vio de Queiroz-Telles (UFPR: Federal University of Paraná)
#2Paula Massaroni Peçanha Pietrobom (UNIFESP: Federal University of São Paulo)
Last.Paulo Mendes Peçanha (UFES: Universidade Federal do Espírito Santo)H-Index: 3
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#1Christelle Bergeron (Université de Sherbrooke)
#2André M. Cantin (Université de Sherbrooke)H-Index: 36
Cystic fibrosis (CF) is a common, life-threatening, multisystemic, autosomal recessive disorder. In the last few years, giant steps have been made with regard to the understanding of CF pathophysiology, allowing the scientific community to propose mechanisms that cause the myriad of CF clinical manifestations. Following the discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989, the structure and function of the CFTR protein were described. Since then, more than...
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#1Senne Cuyx (Katholieke Universiteit Leuven)H-Index: 1
#2K. De Boeck (Katholieke Universiteit Leuven)H-Index: 46
Detailed knowledge of how mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene disturb the trafficking or function of the CFTR protein and the use of high-throughput drug screens have allowed novel therapeutic strategies for cystic fibrosis (CF). The main goal of treatment is slowly but surely shifting from symptomatic management to targeting the underlying CFTR defect to halt disease progression and even to prevent occurrence of CF complications. CFTR potentiators fo...
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#1Carlo Castellani (Istituto Giannina Gaslini)H-Index: 33
#2Barry Linnane (UL: University of Limerick)H-Index: 7
Last.Daniel Peckham (St James's University Hospital)H-Index: 26
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The diagnosis of cystic fibrosis (CF) has traditionally relied on the presence of clinical features of the disease. Today, diagnosis through newborn screening (NBS) is becoming the standard of modern CF care. CF NBS programs can identify CF prior to clinical presentation, but for the advantages of an early diagnosis to accrue a scrupulous system must be in place to ensure all steps in the program are performing. As we move rapidly into the era of CF transmembrane conductance regulator (CFTR) pro...
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Cystic fibrosis (CF) is now more common in adults than children in countries with well-developed health care systems. The number of adults continues to increase and will further increase if the new cystic fibrosis transmembrane conductance regulator (CFTR) modulators are disease modifying. Most of the complex morbidity and almost all the mortality of CF occur in adults and will increasingly follow this pattern even with new effective modulator therapies. Maintaining good quality of life includin...
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Over the past decade, the incidence of nontuberculous mycobacterial (NTM) infection has been increasing in cystic fibrosis patients. Along with this have come a host of complications and burdens to patients that threaten longevity and quality of life. The two main constituents of NTM pulmonary disease, Mycobacterium avium complex (MAC) and M. abscessus, are notoriously difficult to treat with suboptimal clinical responses and are accompanied by high treatment burdens for patients. This review ai...
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