The 2016 International Rare Lung Diseases Clinical Research Conference

Cincinnati Childrens Hosp Med Ctr, Cincinnati OH

Investigators

Abstract

ABSTRACT The 2016 International Rare Lung Diseases Clinical Research Conference (RLDCl?2016) will be the largest and most comprehensive international scientific research meeting focused to rare lung diseases ever held. Remarkable advances in some rare lung diseases have occurred in the past few years, due in large part to the unique ability to understand the pathogenesis of these disorders from the vantage point of a primary molecular defect. For example, recent studies in pulmonary alveolar proteinosis (PAP) have led to novel diagnostic and therapeutic approaches, including the discovery of a new form of the disease, the development of a diagnostic biomarker that is highly specific and sensitive, and a trial demonstrating benefit from inhaled GM-CSF therapy. Lymphangioleiomyomatosis (LAM) has been transformed from a death sentence to a chronic disease with an effective treatment that stabilizes lung function and improves quality of life. These advances have been made possible, in large part, through a series of organized collaborations between the National Institutes of Health, international investigators, clinical care providers and patient advocacy groups. Most other rare lung diseases lack this synergistic approach, however, and are too uncommon to attract the curiosity and dedicated attention of skilled investigators individually. We are convinced that the time for a comprehensive rare lung disease meeting is now, to discuss what is known, share effective approaches, and translate the discoveries of the last three decades to patient benefit. The Rare Lung Diseases Consortium (RLDC), a member of the NIH-supported Rare Diseases Clinical Research Network (RDCRN), is organizing the RLDCl?2016 to address these needs, in support of the Central Hypothesis that a crucial aspect of accelerating the advancement in rare lung disease research is establishing and enhancing communication among patients, clinicians, and scientists. The long term goals of the RLDC include the development of molecular pathway-based diagnostics and therapeutics and translation to help people with rare lung diseases ? in real time. This conference will assemble international leaders, trainees, practicing clinicians, and investigators interested in rare lung diseases and aims to: 1) provide clinicians with information on current state of diagnostics and treatments for rare lung diseases; 2) formulate effective communication platforms to increase connectivity of clinicians and scientists who focus on rare lung diseases; 3) build and enhance a network of rare lung disease clinicians, scientists and patient advocates to support transfer of successful strategies from mature programs to under-represented and less advanced programs; 4) discuss the next best steps for development of molecular pathway-based diagnostics and therapeutics to address the needs of patients with rare lung diseases. RLDCl?2016 will admit scientifically-qualified participants of any sex, race, age, color, religious affiliation and national origin. It will be the first conference in over a decade comprising physician-scientists and patient advocates focused to rare lung disease research.