The FDA has approved BridgeBio Pharma’s acoramidis, branded as Attruby, for transthyretin amyloidosis cardiomyopathy (ATTR-CM), positioning the oral therapy to compete head-to-head with Pfizer’s tafamidis franchise (Vyndamax/Vyndaqel/Vynmac). According to PharmaPhorum.com,…
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative heart disease driven by protein instability. Normally a tetramer, transthyretin can dissociate into monomers that misfold, aggregate, and deposit as amyloid fibrils within…
Diagnosing transthyretin amyloid cardiomyopathy (ATTR-CM) is challenging because its symptoms are vague and overlap with many conditions. Providers typically follow a stepwise approach that combines history-taking, targeted testing, and imaging…
The latest statistics on heart disease and its effect on Black communities are startling. According to an article in the Dallas Examiner, Black people are fifty to seventy percent more…
AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…
According to a recent article, investigators created a machine learning model in order to identify if patients are at risk of rare cardiomyopathy. Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin amyloid cardiomyopathy…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
Pfizer Rare Disease is an approach taken by pharmaceutical giant Pfizer to develop treatments for rare and orphan diseases. In the past, the company has worked with the National Newspaper…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…
Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a rare disease that occurs when amyloids, an…
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