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Goal 2: Reduce Human Disease

Study of Prevention of Airflow Obstruction Progression

There should be a study for prevention of Airflow Obstruction Progression. Rationale: All diseases have a prodrome followed by an early face of disease progression. Complex medical chronic diseases can only be slowed through the addition of several interventions all of which contribute to a final improvement of prognosis (witness CV diseases).
Definition of “Early Disease”: Would be aimed at patients with airflow obstruction... more »

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Goal 2: Reduce Human Disease

Registry of new-onset, post-operative atrial fibrillation

There is a need for an observational registry of new-onset post-operative atrial fibrillation (AF) patients to better understand the mechanisms of post-operative AF and allow clinicians to define patient-specific AF phenotypes and treatments. The registry’s information and infrastructure could be used to develop and conduct clinical trials that provide strong evidence for guidelines in treating this common problem.

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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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Goal 2: Reduce Human Disease

What is the effect of variant genes on AVM development in HHT

Natural genetic variation between individuals can influence the outcome of carrying an HHT mutation. Some gene variants may be protective while others may increase the risk of AVM or telangiectasis. By identifying the variant genes that alter risk of AVM may give clues to the molecular mechanisms of AVM formation and provide new drug targets

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Goal 2: Reduce Human Disease

Cardiovascular dysfunction in geriatric trauma patients

There is too little research funding addressing cardiovascular dysfunction in geriatric trauma patients. There have been little interest in funding this work. Yet, the geriatric population is growing.



Geriatric trauma patients are predominantly women.



Historically, the trauma societies provide guidance for diagnosis and treatment of severe trauma. However, "trauma guidance" historically was the same for children,... more »

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Goal 2: Reduce Human Disease

International collaboration for genetic and metabolic research on specific human population

During recent years, clinical research including well-organized randomized clinical trials in developed countries generated large database and human biological sample banks. These are valuable resources for human disease research. Mechanisms to encourage and facilitate international collaboration for genetic and metabolic research using database and human biological samples from specific human disease population of international... more »

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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 »

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Goal 2: Reduce Human Disease

Development of Optimally Hemostatic, Systemically Safe, Platelet Mimetics or Substitutes

What are the knowledge and technological gaps in production, evaluation and clinical translation of donor-independent platelets for transfusions? Specific questions include: a) How can stem or progenitor cells be expanded to maximize platelet production?; b) What are the hemostatically relevant design and function requirements and evaluation metrics for ideal/optimal “biologic” and “synthetic” platelets? c) What preclinical... more »

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