Key result
Gene discovery over the past 25 years has linked Marfan syndrome and related disorders to TGF-β pathway defects, shifting therapeutic focus from hemodynamic stress management to TGF-β antagonism.
This review highlights the past 25 years of gene discovery in Marfan syndrome and related disorders, emphasizing the critical role of the TGF-β pathway in disease pathogenesis and as a therapeutic target.
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Supports TGF-β antagonism research in Marfan syndrome; leaves open clinical efficacy pending trials.
Verstraeten et al. (2016) conducted a review in Marfan syndrome and related disorders. Gene discovery over the past 25 years has linked Marfan syndrome and related disorders to TGF-β pathway defects, shifting therapeutic focus from hemodynamic stress management to TGF-β antagonism.
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