Goal 2: Reduce Human Disease

To extend our knowledge of the pathobiology of heart, lung, blood, and sleep disorders and enable clinical investigations that advance the prediction, prevention, preemption, treatment, and cures of human disease.
(@craighersh)

Goal 2: Reduce Human Disease

Precision medicine in non-malignant lung diseases

NIH has a major initiative in Precision Medicine, including whole genome sequencing. In contrast to cancer, mutations with large clinical effects are expected to be uncommon in most non-malignant chronic diseases, such as asthma and COPD. Other data types such as gene expression, biomarkers, and micro RNAs must be combined with clinical and imaging phenotyping to advance Precision medicine in non-malignant lung diseases. ...more »

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(@nhlbiforumadministrator)

Goal 2: Reduce Human Disease

Phase III efficacy trials of tuberculosis drugs

1) Phase III efficacy trials of new tuberculosis drugs (e.g., bedaquiline, delamanid, PA-824) that have shown promise in early phase studies for multidrug-resistant tuberculosis. 2) Phase III efficacy trials of new and existing tuberculosis drugs to development very short course regimens (3-4 months). 3) Phase III efficacy trials of new and existing drugs for treatment of latent tuberculosis infection in contacts of ...more »

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(@gcioffi)

Goal 2: Reduce Human Disease

Iron Metabolism in thalassemia syndromes, and other rare and common anemias

Thanks to elegant work since the 1990s, many details of the role of iron regulation and metabolism have been elucidated. Recent efforts in academic and pharmaceutical laboratories aim to translate these discoveries into therapies that may alleviate anemia in iron-refractory states (including anemia of inflammation and congenital disorders), and may have important therapeutic effects in non-transfusion-dependent thalassemia, ...more »

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40 net votes
55 up votes
15 down votes
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(@bcelli)

Goal 2: Reduce Human Disease

COPD and co-morbidities

Society is ageing and chronic degenerative diseases including COPD are increasingly occurring together. The critical question is whether certain diseases occur together by chance or are they occurring together because they share pathobiological commonalities and mechanisms? This leads to a series of practical consequences and questions 1. Which diseases are occurring concurrent with COPD more than chance alone would ...more »

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20 net votes
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(@nhlbiforumadministrator)

Goal 2: Reduce Human Disease

Lung Transplantation

As in other solid organ transplants, adherence to the medical regimen after adult and pediatric lung transplantation is less than ideal and contributes to poor health outcomes. a) How can we improve the prediction of which lung transplant recipients are at greatest risk for nonadherence? b) What are the most efficient and effective ways of assessing nonadherence in the clinical setting? c) Conduct clinical trials of ...more »

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(@jamesr.priestmd)

Goal 2: Reduce Human Disease

Understanding the Genetic & Epigenetic Basis of Congenital Heart Disease?

Over the last thirty years, our fundamental understanding of the genetics and pathogenesis of congenital heart disease has lagged the tremendous advances in the surgical and clinical care of infants with this group of disorders. We need to close this gap with investigation into the genetic basis of congenital heart malformations to develop new models of disease. The goall is translate an improved molecular genetic and ...more »

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22 net votes
37 up votes
15 down votes
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