Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome
| Status: | Withdrawn |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | Any - 24 |
| Updated: | 4/21/2016 |
| Start Date: | April 2011 |
Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular
differentiation, and other functions in most cells. TGF-β levels play a major role in the
pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long
extremities, lens dislocation in the eyes and heart complications such as mitral valve
prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the
underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I,
normally an important component of elastic fibers it has been shown that the Marfan syndrome
phenotype can be relieved by addition of a TGF-β antagonist in affected mice.
differentiation, and other functions in most cells. TGF-β levels play a major role in the
pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long
extremities, lens dislocation in the eyes and heart complications such as mitral valve
prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the
underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I,
normally an important component of elastic fibers it has been shown that the Marfan syndrome
phenotype can be relieved by addition of a TGF-β antagonist in affected mice.
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular
differentiation, and other functions in most cells. TGF-β levels play a major role in the
pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long
extremities, lens dislocation in the eyes and heart complications such as mitral valve
prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the
underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I,
normally an important component of elastic fibers it has been shown that the Marfan syndrome
phenotype can be relieved by addition of a TGF-β antagonist in affected mice. This suggest
that while the symptoms of Marfan syndrome may seem consistent with a connective tissue
disorder, the mechanism is more likely related to reduced sequestration of TGF-β by
fibrillin.
differentiation, and other functions in most cells. TGF-β levels play a major role in the
pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long
extremities, lens dislocation in the eyes and heart complications such as mitral valve
prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the
underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I,
normally an important component of elastic fibers it has been shown that the Marfan syndrome
phenotype can be relieved by addition of a TGF-β antagonist in affected mice. This suggest
that while the symptoms of Marfan syndrome may seem consistent with a connective tissue
disorder, the mechanism is more likely related to reduced sequestration of TGF-β by
fibrillin.
Inclusion Criteria:
- Individual with Marfan syndrome consented in to the Main Atenolol Vs. Losartan NIH
study.
Exclusion Criteria:
- Subjects in the main PHN Marfan trial who have not achieved the maintenance drug
dosing or who have stopped taking study drug.
We found this trial at
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Children's Memorial Hospital, Chicago Ann & Robert H. Lurie Children
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