28th Annual North American Cystic Fibrosis Conference

Oct 9, 2014 ‐ Oct 11, 2014



The synchronized sessions from the 2014 NACFC are NOW AVAILABLE!

Full NACFC registrants have complimentary access to the recordings of the 2014 NACFC Plenary sessions, Symposia and Workshops.

Short Course presentations held on Wednesday, October 8, are also available for an additional fee. To purchase access to the Short Course presentations, click here.


Sessions

S08: CFTR: Gene Editing Strategies for Therapy & Research

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

This session will describe two technologies to manipulate genes such as CFTR for potential therapeutic purposes as well as for more fundamental mechanistic studies about cell biology. One of these technologies is genome editing by the CRISPR/Cas9 system, in which targeted alterations are made in the genome of particular cells. Various applications of the technology, from mutation correction in primary cells to creation of mutations to study a mutation's effects on cellular processes or as a therapeutic tool will be discussed. RNA editing, by which one can correct mutations in messenger RNA without modifying the genomic DNA from which it was transcribed will also be discussed.

Speaker(s):
Standard: $49.99

S09: GMS: The Complex Genetics of CF & Their Functional Effects

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Recent research has identified modifier genes of CF disease severity through genome-wide association studies, linkage analysis studies and candidate gene approaches. This session will describe several studies focusing on how to understand the biological and functional significance of several of these modifier genes.

Speaker(s):
Standard: $49.99

S11: INF/MIC: Multiple Inhaled Antibiotics: Multiple Therapeutic Considerations

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Suppressive antibiotic treatment of CF patients with chronic P. aeruginosa airway infection is the standard of care in Europe and North America. With the recent inhaled aztreonam approval, there are now three antipseudomonal antibiotic classes that are readily available and commonly utilized to manage chronic P. aeruginosa airway infections: polymyxins (colistimethate), aminoglycosides (gentamicin, tobramycin) and beta lactams (aztreonam). These agents have all been studied and developed as monotherapies, but there is growing interest in the use of these agents in various combinations in individual patients to improve outcomes and extend the period of efficacy of suppressive treatment.

Speaker(s):
Standard: $49.99

S12: CLIN: Caroline McPherson Symposium: Care of Infants & Pre-school-aged Children With CF

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Early aggressive treatment of pulmonary disease and optimization of nutritional status in cystic fibrosis during early childhood is essential to achieving improved outcomes in adulthood. Unfortunately, there are several challenges within this age group ranging from the extensive need for parental education, rapid movement through developmental stages and lack of concrete evidence from randomized controlled trials to guide care. This session will address the infant and preschool guidelines in the context of this challenging developmental period of life.

Speaker(s):
Standard: $49.99

S14: NTR: The Relationship of Nutrition to Quality of Life

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

This session will review current knowledge of the relationship of nutrition to Quality of Life; specifically weight, height, body image, supplemental feeding, newborn screening and the feeding relationship, and nutrient intake and depression

Speaker(s):
Standard: $49.99

S10: NT: Fruit of the Pipeline: The Latest Clinical Trial Data

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

This session will address recent clinical trials that test novel approaches to CF therapies directed at overcoming the basic defect of missing or dysfunctional CFTR.

Speaker(s):
Standard: $49.99

W11: CFTR-NT: Overcoming Barriers to Fixing CFTR

Oct 10, 2014 2:00pm ‐ Oct 10, 2014 3:45pm

Correcting mutant CFTR (in particular F508del-CFTR) is a difficult task due to a complex nature of the basic defect. Overcoming the biosynthetic processing arrest, improving defective gating, and increasing the plasma membrane stability are required to fix F508del-CFTR. Several extrinsic factors also limit current rescue strategies. In this session, we will discuss the barriers and present novel approaches to fix mutant CFTR.

Speaker(s):
Standard: $49.99

W12: APP&D: Animal Models of CF Airways Disease

Oct 10, 2014 2:00pm ‐ Oct 10, 2014 3:45pm

Workshop (W12) will highlight animal models that contribute to our understanding of CF airway disease. The workshop will include presentations from four different model species. Each presentation will offer insights into disease mechanisms that can contribute to the development and/or exacerbation of CF airway disease.

Speaker(s):
Standard: $49.99

W13: APP&D: Biomarkers & Endpoints for Clinical Trials

Oct 10, 2014 2:00pm ‐ Oct 10, 2014 3:45pm

One of the greatest challenges in the conduct of CF clinical trials is the designation of clinically relevant endpoints, which are sufficiently rigorous to reflect biological changes and to meet regulatory requirements. This session will include a broad variety of approaches to reporting outcomes in CF, from CFTR function to imaging changes to patient-reported outcomes. The final discussion will highlight current regulatory requirements in the context of outcomes, allowing attendees an understanding of ongoing challenges in the field.

Speaker(s):
Standard: $49.99

W14: NT-CFTR: Beyond Common CFTR Mutations: Leaving No Patient Behind

Oct 10, 2014 2:00pm ‐ Oct 10, 2014 3:45pm

With over 1,000 known CFTR mutations, which are associated with varying symptoms and severity of disease, identifying the best approaches to treat different mutations remains challenging. This workshop will focus on the CFTR defects caused by rare mutations and the strategies to understand and mitigate the defects caused by them. The speakers will present: a) basic research using biophysical, biochemical and electrophysiological approaches to assess the response of mutant CFTR to correctors; b) translational studies where approaches using patient derived cell models for personalized medicine will be discussed; and c) clinical research studies employing Ivacaftor for rare CFTR mutations.

Speaker(s):
Standard: $49.99
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