Goal 3: Advance Translational Research

Submitted by (@sicklecellwarrior)

Dissemination & Implementation of new treatments and therapies in sickle cell disease

Are current advances in gene editing, new drug therapies and less restrictive BMT criteria being explained and rolled out to the sickle cell community in an effective and timely manner? When can people living with sickle cell disease experience a better quality of life on more permanent based on the treatments we already have?

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46 net votes
55 up votes
9 down votes
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Goal 2: Reduce Human Disease

Submitted by (@nhlbiforumadministrator1)

A Program of Research in the Prevention of Chronic Heart Failure

There is a need to address chronic heart failure (HF) through improved identification of patients at risk for HF and of patients with pathological ventricular remodeling who have minimal evidence of clinical HF, and more focused and individualized pharmacologic and lifestyle treatments and monitoring of patients with HF risk. Approaches would include big data collection, omics, statistical modeling, and focused clinical ...more »

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17 net votes
28 up votes
11 down votes
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Goal 3: Advance Translational Research

Submitted by (@njkenyon)

Using "omics" technologies to define responders to drug therapies

Metabolomic and proteomic technologies open tremendous avenues to define at the systemic level and, in the case of the lung, the organ level response to drug and non-drug interventions. The concept of responders and non-responders to therapies is poorly defined and hampers development of biomarkers and appropriate animal models. Omics technologies can bridge these important areas. In lung disease, breath analysis could ...more »

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7 net votes
13 up votes
6 down votes
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Goal 3: Advance Translational Research

Submitted by (@mtothbsf)

Substituting scientific-medical insight before profit in drug development

Since the Pure Food and Drug Act of 1906 and the rise of the FDA, the US federal government has directly inserted itself into medical research, primarily from a business perspective. The Orphan Drug Act of 1983 monetarily incentivized the process for rare diseases, but for ultra-rare diseases of < 1,000 patients, it does not work. Successful drugs for rare diseases have enormous price tags to compensate their development ...more »

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6 net votes
11 up votes
5 down votes
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Goal 4: Develop Workforce and Resources

Submitted by (@mallampallirk)

Training in Small Molecule Discovery and Development

The current generation of heart,lung, and blood investigators are not equipped with competitive training needed to approach, design, and test appropriately small molecule therapeutics that may move through the FDA pipeline. Appropriate in silico ligan or structure based design, HTS, design of Pd/Pk models, "go-no-go" branchpoints in drug development, screening approaches, and drug target validation are issues that are ...more »

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3 net votes
10 up votes
7 down votes
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