(@daniel.perez)

Goal 3: Advance Translational Research

Does non-invasive ventilation (Bi-Pap) improve quality of life and muscle health in FSHD patients

What is the difference in quality of life and functioning in adult facioscapulohumeral muscular dystrophy patients who are on non-invasive ventilation Bi-Pap versus age matched FSHD patients not on ventilation in a long term longitudinal study?

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(@daniel.perez)

Goal 2: Reduce Human Disease

What causes variation in severity of skeletal muscle, lung, pulmonary and heart system symptoms in FSHD muscular dystrophy

Loss of diaphragm function and impaired respiration is a leading driver of morbidity and mortality in the adult muscular dystrophies such as facioscapulohumeral muscular dystrophy, and therefore requires additional study

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-10 net votes
6 up votes
16 down votes
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