Is FVC a valid surrogate for mortality in patients with idiopathic pulmonary disease?
Does prednisone therapy improve outcomes in acute exacerbation of IPF?
Do the non-pharmacological interventions of pulmonary rehabilitation and supplemental oxygen for exertional hypoxemia improve quality of life and functional status in patients with chronic fibrotic ILD?
What is the natural history of the best characterized ChILD disorders (surfactantrelated sequence variants, neuroendocrine cell hyperplasia of infancy (NEHI),pulmonary interstitial glycogenosis (PIG),idiopathic pulmonary hemosiderosis)?
What is the relationship of ChILD disorders to adult diffuse lung disease?
What is the relationship of ChILD disorders to more common childhood respiratory diseases?
What is the relationship of ChILD disorders to other clinical populations that manifest ILD?
Does "goal-targeted" therapy (with adjustments/additional therapy, if certain "goals" are not achieved) improve quality of life, functional status, and survival in patients with pulmonary arterial hypertension? Trials of therapies for hepatopulmonary syndrome.
Does pulmonary rehabilitation or regular exercise improve outcomes in patients with PVD?
NHLBI should engage more interactively with the Office of Rare Diseases (ORD).
NHLBI should establish a rare disease biorepository that can be used by PhD and MD scientists, and propose new therapies based on the insights gained.
Do SCD patients with hemodynamics consistent with pulmonary arterial hypertension (PAH) respond to medications designed to treat PAH?
A study section should be seated for clinical trials on rare disease. Members of this study section should consist only of individuals who have previously performed phase I and/or phase II trials, developed IND or IDE applications, or who have extensive experience in informatic or biometric support for clinical trials. My opinion is that seating individuals on these sections who have a laboratory career in cellular or... more »
What is the contribution of airways disease to acute and chronic pulmonary distress?
NHLBI should establish a rare disease registry.