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.
Goal 2: Reduce Human Disease
Does pulmonary rehabilitation or regular exercise improve outcomes in patients with PVD?
To conduct longitudinal gene expression analysis of adaptive immunophenotypes within newly-diagnosed sarcoidosis subjects who spontaneously resolve their pulmonary disease compared to those to experience disease progression.
To delineate if sarcoidosis pulmonary and extrapulmonary disease reflect the same immunophenotypes.
To investigate the effects of antimycobacterial therapy on pulmonary and extrapulmonary immune response and granulomatous disease.
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?
We also wish to draw attention to the rise in the prevalence of hypertension in the pediatric population, mostly as a consequence of the childhood obesity epidemic.
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?
Effect of adenotonsillectomy on behavioral and cardiovascular outcomes in children with primary snoring
Effect of adenotonsillectomy on neurocognitive and behavioral outcomes in infants and and toddlers with obstructive sleep apnea syndrome.
Effectiveness of medications for cataplexy in children.
NHLBI should establish a rare disease registry.