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
Cystic Fibrosis in Focus
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Podcast Overview
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
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Publishing Since
8/1/2016
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Recent Episodes
February 6, 2020
An In-Depth Look at the Evolution of Cystic Fibrosis Treatments
Host: Paul Doghramji, MD <br> Guest: Gregory S. Sawicki, MD, MPH <br> <p>Even though there currently isn’t a cure for cystic fibrosis, there may finally be hope on the horizon for the more than 70,000 people around the world who are living with this debilitating disease. Joining Dr. Paul Dogramji to talk about the emerging treatment options for cystic fibrosis is Dr. Gregory Sawicki, Director at the Cystic Fibrosis Center and Assistant Professor of Pediatrics at Harvard Medical School. </p>
August 1, 2016
Journal of Cystic Fibrosis: Efficacy and Safety of Levofloxacin Inhalation Solution (APT-1026) in Stable Cystic Fibrosis Patients
<h1>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.</h1> <h2>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.</h2> <p><a href="https://www.ncbi.nlm.nih.gov/pubmed/26852040" target="_blank"><strong><span style="text-decoration: underline;">J Cyst Fibros. 2016 Jul;15(4):495-502. doi: 10.1016/j.jcf.2015.12.004. Epub 2016 Feb 4.</span></strong></a></p> <h3><span style="font-size: 1.17em;">Abstract</span></h3> <p><strong>RATIONALE:</strong></p> <p>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.</p> <p><strong>OBJECTIVES:</strong></p> <p>To compare the safety and efficacy of a 28-day course of treatment with LIS 240mg or placebo BID in persons ≥12years old with CF and chronic P. aeruginosa infection.</p> <p><strong>METHODS:</strong></p> <p>A multinational, randomized (2:1), double-blinded study of LIS and placebo over 28days in CF patients ≥12years with chronic P. aeruginosa infection. Time to exacerbation was the primary endpoint. FEV1 (% predicted) and patient-reported …
August 1, 2016
Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target
<p><strong>A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR.</strong></p> <p>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.</p> <p><a href="https://www.ncbi.nlm.nih.gov/pubmed/27035618" target="_blank"><strong><span style="text-decoration: underline;">Cell Death Differ. 2016 Aug;23(8):1380-93. doi: 10.1038/cdd.2016.22. Epub 2016 Apr 1.</span></strong></a></p> <p><strong>Abstract</strong><br/>We 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 such mice were autophagy-competent. Primary nasal cells from patients bearing different class II CFTR mutations, either in homozygous or compound heterozygous form, responded to the treatment in vitro. We assessed individual responses to cysteamine plus EGCG in a single-centre, open-label phase-2 trial. The combination treatment decreased sweat chloride from baseline, increased both CFTR …
4 total episodes available
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