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.

Goal 2: Reduce Human Disease

Clinical trial of AAT augmentation therapy in PI MZ subjects

Although severely alpha-1 antitrypsin deficiency (PI ZZ) individuals are rare, PI MZ subjects comprise approximately 3% of the US population. Mounting evidence suggests that PI MZ is a significant risk factor for COPD. A clinical trial of COPD exacerbation frequency using AAT augmentation therapy in PI MZ subjects with moderate-to-severe COPD could provide a rationale for AAT augmentation therapy (or small molecule neutrophil ...more »

Submitted by (@nhlbiforumadministrator)

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Goal 2: Reduce Human Disease

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently ...more »

Submitted by (@katherinek)

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Goal 2: Reduce Human Disease

Moonshot: Turning the BMT EMR into a Research Record

The critical challenge is to develop a standards-based BMT electronic medical record (EMR) and integrate research capacity into the architecture of EMR systems. The ultimate goal would be to build de-identified complete data-sets which can be used to support observational studies and clinical trials, improve transplant outcomes and inform public policy.

Submitted by (@rakeshgoyal)

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Goal 2: Reduce Human Disease

What are the biological consequences of sleep loss or disruption and how can they best be avoided?

Arousals in obstructive sleep apena (OSA) are life saving, but the associated disruption of sleep is now thought to cause cognitive impairment, increased risk of high blood pressure and atherosclerosis, as well as glucose intolerance and metabolic syndrome. The mechanisms for these downstream effects, however, are not well understood. Can these specific pathophysiological mechanisms be identified, and can ways for mitigating ...more »

Submitted by (@csaper)

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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 »

Submitted by (@craighersh)

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Goal 2: Reduce Human Disease

HEALTH CARE DISPARITIES IN DIAGNOSIS AND TREATMENT OF COMMON SLEEP AND CIRCADIAN DISORDERS

There is evidence of a higher prevalence of sleep and circadian disorders in different ethnic groups. This is true for both adult and pediatric subjects. There is also evidence that minority populations in lower socioeconomic groups do not seek evaluation for sleep disorders as frequently as other segments of our population. There is also evidence that they are less adherent to treatments such as nasal CPAP for obstructive ...more »

Submitted by (@jnoel0)

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Goal 2: Reduce Human Disease

Treatment Options for Diabetics and Impact on Cardiovascular Health

As a clinician, over the years I have noted major differences in adverse cardiovascular outcomes in diabetics who are treated with insulin +/- oral agents compared to those only treated with oral agents. Cardiovascular events occur much less often and at a much later timeframe in diabetics ("Type 2/adult onset") treated with insulin as the primary method. Even with newer agents, there may be slight improvement, but ...more »

Submitted by (@patty.gladowski)

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Goal 2: Reduce Human Disease

Condom with Retention Means And Shield (Patent Pending)

Condom designed to protect a males pubic areas and scrotum, fingers and hands from coming in contact with infectious fluids. The adoption of a shield to reduce the occurrence of HPV and other STI infections. This is the first and only condom in the world that uses a latex shield which is designed to protect a promiscuous males fingers and hands from HPV and other infectious human secretions that may be in fluids,. We ...more »

Submitted by (@alexandercan.aw)

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