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Cystic Fibrosis in Focus

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Cystic Fibrosis in Focus
Cystic Fibrosis in Focus

Cystic Fibrosis in Focus

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Plays
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About Us

Cystic fibrosis (CF) is one of the most prevalent global congenital disorders, affecting some 70,000 individuals worldwide and 30,000 children and adults in the United States. While clinical efforts are expanding worldwide to improve diagnostic testing and treatment approaches for this progressive lung disease, targeted therapies for CF against any specific mutation remain elusive.The Cystic Fibrosis in Focus series gathers the latest information from leading medical experts devoted to advancing knowledge in pursuit of treating patients with CF. With education spanning topics such as pinpointing CF's underlying genetic basis, identifying specific mutations, improving clinical detection methods, and providing more targeted therapies, The Cystic Fibrosis in Focus series delivers the critical tools needed to improve care for CF patients.​In Partnership with

Latest Episodes

Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR. Tosco A, De Gregorio F, Esposito S, De Stefano D, Sana I, Ferrari E, Sepe A, Salvadori L, Buonpensiero P, Di Pasqua A, Grassia R, Leone CA,Guido S, De Rosa G, Lusa S, Bona G, Stoll G, Maiuri MC, Mehta A, Kroemer G, Maiuri L, Raia V. Cell Death Differ. 2016 Aug;23(8):1380-93. doi: 10.1038/cdd.2016.22. Epub 2016 Apr 1. AbstractWe previously reported that the combination of two safe proteostasis regulators, cysteamine andepigallocatechin gallate (EGCG), can be used to improve deficient expression of the cystic fibrosistransmembrane conductance regulator (CFTR) in patients homozygous for the CFTR Phe508del mutation. Here we provide the proof-of-concept that this combination treatment restored CFTR function and reduced lung inflammation (P<0.001) in Phe508del/Phe508del or Phe508del/null-Cftr (but not in Cftr-null mice), provided that...

-2 s2016 AUG 1
Comments
Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

Journal of Cystic Fibrosis: Microbial Colonization and Lung Function in Adolescents With Cystic Fibrosis

Microbial colonization and lung function in adolescents with cystic fibrosis. Hector A, Kirn T, Ralhan A, Graepler-Mainka U, Berenbrinker S, Riethmueller J, Hogardt M, Wagner M, Pfleger A, Autenrieth I, Kappler M, Griese M, Eber E, Martus P, Hartl D. J Cyst Fibros. 2016 May;15(3):340-9. doi: 10.1016/j.jcf.2016.01.004. Epub 2016 Feb 5. Abstract With intensified antibiotic therapy and longer survival, patients with cystic fibrosis (CF) are colonized with a more complex pattern ofbacteria and fungi. However, the clinical relevance of these emerging pathogens for lung function remains poorly defined. The aim ofthis study was to assess the association of bacterial and fungal colonization patterns with lung function in adolescent patients with CF. Microbial colonization patterns and lung function parameters were assessed in 770 adolescent European (German/Austrian) CF patients in a retrospective study (median follow-up time: 10years). Colonization with Pseudomonas aeruginosa and MRSA were...

2 MIN2016 AUG 1
Comments
Journal of Cystic Fibrosis: Microbial Colonization and Lung Function in Adolescents With Cystic Fibrosis

Journal of Cystic Fibrosis: Efficacy and Safety of Levofloxacin Inhalation Solution (APT-1026) in Stable Cystic Fibrosis Patients

A phase 3, multi-center, multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of levofloxacin inhalation solution (APT-1026) in stable cystic fibrosis patients. Flume PA, VanDevanter DR, Morgan EE, Dudley MN, Loutit JS, Bell SC, Kerem E, Fischer R, Smyth AR, Aaron SD, Conrad D, Geller DE,Elborn JS. J Cyst Fibros. 2016 Jul;15(4):495-502. doi: 10.1016/j.jcf.2015.12.004. Epub 2016 Feb 4. Abstract RATIONALE: For patients with cystic fibrosis (CF), the use of inhaled antibiotics has become standard of care to suppress chronic Pseudomonas airways infection. There are limited antibiotic options formulated and approved for inhaled use and antibiotic efficacies attenuate over time, making additional inhaled antibiotic classes desirable. APT-1026 (levofloxacin inhalation solution, LIS) is a fluoroquinolone in development for management of chronic P. aeruginosa airways infection in patients with CF. OBJECTIVES: To compare the safety and efficacy...

3 MIN2016 AUG 1
Comments
Journal of Cystic Fibrosis: Efficacy and Safety of Levofloxacin Inhalation Solution (APT-1026) in Stable Cystic Fibrosis Patients
the END

Latest Episodes

Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR. Tosco A, De Gregorio F, Esposito S, De Stefano D, Sana I, Ferrari E, Sepe A, Salvadori L, Buonpensiero P, Di Pasqua A, Grassia R, Leone CA,Guido S, De Rosa G, Lusa S, Bona G, Stoll G, Maiuri MC, Mehta A, Kroemer G, Maiuri L, Raia V. Cell Death Differ. 2016 Aug;23(8):1380-93. doi: 10.1038/cdd.2016.22. Epub 2016 Apr 1. AbstractWe previously reported that the combination of two safe proteostasis regulators, cysteamine andepigallocatechin gallate (EGCG), can be used to improve deficient expression of the cystic fibrosistransmembrane conductance regulator (CFTR) in patients homozygous for the CFTR Phe508del mutation. Here we provide the proof-of-concept that this combination treatment restored CFTR function and reduced lung inflammation (P<0.001) in Phe508del/Phe508del or Phe508del/null-Cftr (but not in Cftr-null mice), provided that...

-2 s2016 AUG 1
Comments
Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

Journal of Cystic Fibrosis: Microbial Colonization and Lung Function in Adolescents With Cystic Fibrosis

Microbial colonization and lung function in adolescents with cystic fibrosis. Hector A, Kirn T, Ralhan A, Graepler-Mainka U, Berenbrinker S, Riethmueller J, Hogardt M, Wagner M, Pfleger A, Autenrieth I, Kappler M, Griese M, Eber E, Martus P, Hartl D. J Cyst Fibros. 2016 May;15(3):340-9. doi: 10.1016/j.jcf.2016.01.004. Epub 2016 Feb 5. Abstract With intensified antibiotic therapy and longer survival, patients with cystic fibrosis (CF) are colonized with a more complex pattern ofbacteria and fungi. However, the clinical relevance of these emerging pathogens for lung function remains poorly defined. The aim ofthis study was to assess the association of bacterial and fungal colonization patterns with lung function in adolescent patients with CF. Microbial colonization patterns and lung function parameters were assessed in 770 adolescent European (German/Austrian) CF patients in a retrospective study (median follow-up time: 10years). Colonization with Pseudomonas aeruginosa and MRSA were...

2 MIN2016 AUG 1
Comments
Journal of Cystic Fibrosis: Microbial Colonization and Lung Function in Adolescents With Cystic Fibrosis

Journal of Cystic Fibrosis: Efficacy and Safety of Levofloxacin Inhalation Solution (APT-1026) in Stable Cystic Fibrosis Patients

A phase 3, multi-center, multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of levofloxacin inhalation solution (APT-1026) in stable cystic fibrosis patients. Flume PA, VanDevanter DR, Morgan EE, Dudley MN, Loutit JS, Bell SC, Kerem E, Fischer R, Smyth AR, Aaron SD, Conrad D, Geller DE,Elborn JS. J Cyst Fibros. 2016 Jul;15(4):495-502. doi: 10.1016/j.jcf.2015.12.004. Epub 2016 Feb 4. Abstract RATIONALE: For patients with cystic fibrosis (CF), the use of inhaled antibiotics has become standard of care to suppress chronic Pseudomonas airways infection. There are limited antibiotic options formulated and approved for inhaled use and antibiotic efficacies attenuate over time, making additional inhaled antibiotic classes desirable. APT-1026 (levofloxacin inhalation solution, LIS) is a fluoroquinolone in development for management of chronic P. aeruginosa airways infection in patients with CF. OBJECTIVES: To compare the safety and efficacy...

3 MIN2016 AUG 1
Comments
Journal of Cystic Fibrosis: Efficacy and Safety of Levofloxacin Inhalation Solution (APT-1026) in Stable Cystic Fibrosis Patients
the END