(@daniel.perez)

Goal 3: Advance Translational Research

Develop guidelines, standard of care, new technologies for respiratory care for adult facioscapulohumeral muscular dystrophy

There is a need for NHLBI to develop guidelines, standard of care, new technologies for respiratory care for adult muscular dystrophy (facioscapulohumeral, myotonic and limb girdle) patients with undiagnosed or unforeseen hypercarbia CO2 retention in the acute setting who end up in trouble to help the families, doctors and patients navigate their way back to stable condition e.g. perhaps going forward with non-invasive ...more »

Voting

-12 net votes
2 up votes
14 down votes
Active
(@makeb0)

Goal 2: Reduce Human Disease

Pulmonary rehabilitation

Can pulmonary rehabilitation change the course of COPD? Is pulmonary rehabilitation a disease-modifying therapy? Does pulmonary rehabilitation improve survival? Does pulmonary rehabilitation reduce readmissions?

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20 net votes
26 up votes
6 down votes
Active
(@katherinek)

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 »

Voting

66 net votes
76 up votes
10 down votes
Active
(@dappell)

Goal 2: Reduce Human Disease

Does the genetic cause of pulmonary fibrosis have implications for treatment response?

Is pulmonary fibrosis due to genetic/heritable causes different than 'sporadic' 'idiopathic' pulmonary fibrosis? With findings in genetics and the emphasis on precision medicine the issue of pulmonary fibrosis being idiopathic will diminish with time. Treatment for this fatal disease should be accessible to those who have pulmonary fibrosis with no other alternatives.

Voting

1 net vote
2 up votes
1 down votes
Active
(@greg.martin)

Goal 2: Reduce Human Disease

Long-term pulmonary function in survivors of critical illness

Pulmonary function is known to suffer during the early recovery phases from critical illness, but the long-term patterns of recovery and associated consequences are uncertain. In addition, the clinical and molecular determinants of progressive deterioration or recovery of pulmonary function remain unknown.

Voting

4 net votes
7 up votes
3 down votes
Active
(@bradley.richmond)

Goal 2: Reduce Human Disease

How can we better understand regional tissue heterogeneity in lung disease?

Many lung diseases (IPF, COPD) are characterized by marked heterogeneity at the tissue level. Unfortunately, most of the tools we currently employ to understand lung disease are unable to elucidate the mechanisms that result in regional heterogeneity. Clinical studies and animal models, while invaluable, generally assume that all lung tissue is similarly affected based on the presence or absence of diagnostic criteria ...more »

Voting

9 net votes
26 up votes
17 down votes
Active
(@ghunninghake)

Goal 2: Reduce Human Disease

Identifying Early Stages of Pulmonary Fibrosis

The majority of translational research designed to improve the lives of patients with pulmonary fibrosis has focused on studies of patients with advanced fibrotic lung disease. In contrast, little effort has been paid to understanding the natural history of pulmonary fibrosis, exploring the mechanisms/pathogenesis of the development of pulmonary fibrosis, and considering work designed to prevent the development of lung ...more »

Voting

29 net votes
35 up votes
6 down votes
Active
(@katherinek)

Goal 2: Reduce Human Disease

Development of right ventricular-targeted therapies in pulmonary arterial hypertension (PAH)

Pulmonary arterial hypertension (PAH) is a complex, progressive condition characterized by high blood pressure in the lungs and restriction of flow through the pulmonary arterial system. A great increase in the treatment armamentarium has been noted for this rare disease in the past 20 years, with 12 new PAH-targeted therapies. Though these therapies do improve cardiac performance, this is most likely due to their primary ...more »

Voting

66 net votes
75 up votes
9 down votes
Active
(@yyzhao)

Goal 4: Develop Workforce and Resources

Establishment of an independent study section on Pulmonary Vascular Biology and Translational Research

The research on pulmonary vascular biology including smooth muscle cell biology and endothelial cell biology and related pulmonary vascular diseases such as pulmonary hypertension and related right heart failure, and endothelial dysfunction in lung vascular inflammation and acute lung injury, as well as pulmonary embolism and lung transplantation has been rapidly expanding. The field is attracting an ever increasing ...more »

Voting

23 net votes
50 up votes
27 down votes
Active
(@katherinek)

Goal 2: Reduce Human Disease

Would patients with pulmonary arterial hypertension (PAH) benefit from background anticoagulation in addition to their PAH-targe

Pulmonary hypertension (PH) is a complex, progressive condition characterized by high blood pressure in the lungs. For several decades, oral anticoagulation has been recommended by some societies for patients with a specific form of PH called pulmonary arterial hypertension. However, the evidence currently supporting this recommendation is very limited. To date, no prospective randomized clinical trial has been completed ...more »

Voting

62 net votes
68 up votes
6 down votes
Active
(@daniel.perez)

Goal 2: Reduce Human Disease

Developing Standards of Care for adult muscular dystrophy (FSHD, DM) patients affected by hypercarbic respiratory insufficiency

There is an unmet need for the NHLBI to foster basic, preclinical and clinical research on the pulmonary consequences of respiratory insufficiency, and specifically with hypercarbic (high CO2) respiratory insufficiency, in facioscapulohumeral muscular dystrophy (FSHD) and other adult muscular dystrophies. The adult muscular dystrophies have received insufficient attention, both from research and clinical practice perspectives. ...more »

Voting

-11 net votes
4 up votes
15 down votes
Active