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Thursday, October 17, 2013
Plenary I - Restoring CFTR Function: Roadmap to a Cure (Part 1)
This session will reivew the essential functions of CFTR in health and the consequences of CFTR mutations. The question, "how much CFTR function is enough to fully restore health," will be discussed. Lessons learned from clinical studies of CFTR modulators in CF patients will be reviewed, along with a vision for the path we must follow to reach our goal of a cure.
Scott H. Donaldson, M.D.
Friday, October 18, 2013
Plenary II - Roadmap to a Cure (Part 2): Clinical Research Pathway to Ensure that All Patients with CF Benefit from Novel Therapies
The session reviews how identifying the genetic mutations in CF is leading to "personalized" approaches to care. Current studies in several mutational classes, as well as non-mutational dependent approaches -- such as gene therapy -- will be reviewed. In addition, clinical trials directed to therapies treating the symptoms associated with CF will be discussed, including antimicrobials, anti-inflammatory approaches and therapies to improve airway hydration and clearance. The session will conclude with a discussion of the next steps -- both opportunities and challenges -- to ensure that all patients with CF benefit from novel therapies.
Bonnie W. Ramsey, M.D.
Saturday, October 19, 2013
Plenary III - CF Advisory Board Track: CFRD: From Bench to Bedside & Back Again
Cystic fibrosis-related diabetes is a common co-morbidity in CF that is associated with worse nutritional status, worse pulmonary function, and decreased survival. Early detection and treatment of CFRD can positively impact these outcomes. One purpose of this session is to review current guidelines for early detection of CFRD and clinical trials demonstrating the impact of treating insulin secretion defects and hyperglycemia in CF. This session also will review the use of new CF animal models to dissect the multi-organ molecular basis of CFTR-dependent abnormalities in glucose and insulin regulation in CF. Lessons learned from these animal models are informing clinical trials on the early pathogenesis of CFRD and the design of appropriate treatments.