(@ctong0)

Goal 2: Reduce Human Disease

Heart Failure with Preserved Ejection Fraction Needs better understanding

Effective treatment for Heart Failure with Preserved Ejection Fraction (HFpEF) currently does not exist. Lack of understanding of underlying mechanism(s) probably contributed to this lack of treatment. The well studied neural-hormonal blockade will not work for HFpEF because down stream kinase targets of adrenergic stimulation enhances myocardial relaxation. Consequently, sustain research outside current main stream thinking... more »

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2 net votes
13 up votes
11 down votes
Active
(@dkass0)

Goal 2: Reduce Human Disease

Heart Failure Therapies

We need much more support for critical basic research to understand and develop transformative therapies for this enormous health care burden. This is not simply a question of epidemiology and large multicenter population data bases. We really need hard core science. It is impossible to know where the next breakthrough will come, and setting aside funds for hot button things - stem cells, or iPS, or gene editing per... more »

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6 net votes
18 up votes
12 down votes
Active
(@gcioffi)

Goal 2: Reduce Human Disease

Combination Iron Chelator Trials in Thalassemia and other transfusion-dependent anemias

Three chelators are presently available in the US and much of the world: parenteral deferoxamine, and oral deferasirox, as well as oral deferiprone. Monotherapy is unsuccessful in a significant minority of patients, due to side effects or inadequate response at tolerable doses. Taking a page from enormously successful strategies for combination oral therapy in hypertension, led by NHLBI and others over the past four... more »

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39 net votes
54 up votes
15 down votes
Active
(@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
Active
(@gcioffi)

Goal 2: Reduce Human Disease

Identifying Chronic Pain and exploring the onset and severity among patients with thalassemia

While the primary pathophysiology of thalassemia is related to globin gene mutations and unbalanced globin chain expression, the downstream consequences are manifold. Chronic pain turns out to be one of the most important factors identified by patients with transfusion dependent thalassemia major in health-related quality of life surveys and patient-reported outcome measures. Even as therapies aimed and gene editing,... more »

Voting

37 net votes
47 up votes
10 down votes
Active
(@josephzasadzinski)

Goal 2: Reduce Human Disease

Sex and Race Differences in Replacement Lung Surfactant Efficacy

Baby boys and girls can respond quite differently to replacement lung surfactants given in response to neonatal respiratory distress syndrome. There are also variations in efficacy between babies of different races, in addition to the generally increased risk of NRDS in minority populations overall to lack of prenatal care or access to neonatal intensive care units. Increasing the availability of surfactant treatment... more »

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-7 net votes
3 up votes
10 down votes
Active
(@jennifergrobinson)

Goal 2: Reduce Human Disease

How can we implement what we already know for ASCVD prevention?

We have a number of highly effective evidence-based interventions that have been shown to reduce ASCVD events - statins, BP drugs, aspirin, acute care. Yet large proportions of high risk population groups are not taking evidence-based treatment. Numerous interventions have been tried at multiple levels from the individual patient to the federal government payors. What are the best practices? How can we systematically... more »

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-9 net votes
3 up votes
12 down votes
Active
(@rakeshgoyal)

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.

Voting

57 net votes
71 up votes
14 down votes
Active
(@jiang001)

Goal 2: Reduce Human Disease

What about the impact of regulation of genes in response to external stimulation on human health

We are focusing a lot on the genes that may be protective or harmful to our lives. But what about the regulation of genes in response to external stimulations, such as psychosocial and/or environmental, that are probably more accountable for whether we live healthier or not.

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-4 net votes
5 up votes
9 down votes
Active
(@nhlbiforumadministrator1)

Goal 2: Reduce Human Disease

Do we yet know all of the Resident Cellular Components of the Human Lung?

The DLD, NHLBI workshops conducted and published (Reference 1, 2, 3) had as their purpose to stimulate research that would identify still obscure or novel cellular components of the human lung to determine cell function in promoting respiratory tract development and in health that contributes to disease, so that better therapy might result. With robust technologies now available, especially genomic advances, how much... more »

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-5 net votes
8 up votes
13 down votes
Active
(@ben.croker)

Goal 2: Reduce Human Disease

Prevent cytopenia in septic patients

Sepsis is the leading cause of death in critically ill patients in the USA, affecting particularly young children and the elderly. A hallmark of septic shock patients upon diagnosis is peripheral blood cytopenia. This persistent cytopenia commonly affects myeloid, lymphoid and erythroid lineages resulting in immunosuppression and is a well-established predictor of fatal outcome. Clinical trials targeting the production... more »

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0 net votes
11 up votes
11 down votes
Active
(@savebasicresearch)

Goal 2: Reduce Human Disease

Optimize treatment strategies for cardiovascular diseases by considering comorbid conditions

Patients seen by doctors often relay a specific health issue and are treated for that particular complaint using a standard list of treatment regimens that do not consider comorbid conditions as a significant beneficial means to optimize positive outcomes. Other than allergy considerations, immediate cost often appears to drive treatments.

Voting

18 net votes
28 up votes
10 down votes
Active
(@hjoffe)

Goal 2: Reduce Human Disease

Research priorities: Sleep disorders in women

Women are more likely to have insomnia and they develop the condition at an earlier age than men. Hormone changes and menopause are two key factors but little is known about the mechanisms, which will inform the treatment. This is an understudied area of scientific focus with vast potential public health impact.

Voting

45 net votes
57 up votes
12 down votes
Active