Phase I trial demonstrates first pharmacological treatment able to improve cardiac function in stiff-heart syndrome nejm
. The study was led by Dr. Pablo Garcia-Pavía, who heads the Inherited Cardiac Diseases Section at Hospital Universitario Puerta de Hierro and is a research scientist at the Centro Nacional de Investigaciones Cardiovasculares and within the Spanish cardiovascular research network .
The study represents a major advance in the treatment of the disease. Although currently available treatments effectively prevent the accumulation of more amyloid fibrils and delay disease progression, they do not directly remove any amyloid protein already deposited in the heart. The only drug approved to treat transthyretin-related cardiac amyloidosis is tafamidis, an oral transthyretin stabilizer. Tafamidis improves survival and reduces hospitalizations; however, it does not reverse disease symptoms that are already established.
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