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Wild-type

Wild-type ATTR is also referred to as ATTRwt. It is not caused by any known genetic mutations, such as in the case of hereditary forms of the disease (hATTR). This disease used to be called SSA or SCA, which stood for Senile Systemic Amyloidosis and Senile Cardiac Amyloidosis, respectively, which are now outdated terminologies. The disease is not known to be directly related to dementia, but it is related to aging.

Deposits of TTR amyloid can be found throughout the body, so it is a systemic amyloidosis disease.  The most common place it is found is in the heart. Wild-type ATTR is also known to cause some cases of carpal tunnel syndrome, which can be the first (early) symptom. Recent data suggests that lumbar spine involvement as well as a rupture of the biceps tendon in the forearm can precede cardiac involvement by many years.

This is a disease that has traditionally been found mostly in men, originally reported in those aged 80 and over. As research continues, wild-type ATTR has been increasingly found in individuals in their early 60s. It is often overlooked as an amyloidosis disease because so many people experience heart problems in their later years.

As with hereditary forms of the disease (hATTR) wild-type ATTR causes problems due to the breaking apart, misfolding and deposition of amyloid protein fibrils in healthy tissue. “Wild-type” refers to this form of the disease because it is the natural form of this protein, without genetic mutation. These deposits can interfere with the heart’s normal function, by causing stiffness of the heart tissue, making it more difficult for the heart to fill, leading to heart rhythm problems and heart failure.

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ATTR-ACT Study Update

On August 6th, 2015, the Phase 3 Transthyretin Cardiomyopathy Study (“ATTR-ACT Study”, B3461028) reached an important milestone by completing enrollment. ATTR-ACT is a multicenter, international, double-blind, placebo-controlled, randomized study to evaluate the efficacy, safety, and tolerability of…

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FDA Approves Two Treatments for hATTR Amyloidosis in 2018

 by Frederick L. Ruberg, MD – Boston University, BUMC, Amyloidosis Center What a momentous Summer of 2018 it has been for transthyretin (ATTR) amyloidosis. First, in July 2018, in the same issue of the New England Journal of Medicine, two different phase 3 clinical trials were reported demonstrating…

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Research

We invest in outstanding scientific research and innovative research models to expedite promising therapies to patients.

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Life Is What You Make It

I am Len Strickland and I was diagnosed with Hereditary Amyloidosis (ATTR) with a variant of Val122ile. There are over 100 different variants of ATTR based on culture and ethnicity. I learned of my affliction at the Mayo Clinic in Rochester, Minnesota on March 29 of 2007. Hereditary Amyloidosis (ATT…

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