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SSA or SCA, which stood for Senile Systemic Amyloidosis and Senile Cardiac Amyloidosis, respectively, which are now outdated terminologies.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 awareness of the disease increases, wild-type ATTR average age at diagnosis is 75. 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.more
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…more
Intellia Therapeutics Receives Authorization to Initiate Phase 1 Clinical Trial of NTLA-2001 for Transthyretin Amyloidosis (ATTR)
Oct 19, 2020 NTLA-2001: First single-course therapy that potentially halts and reverses ATTR On track to dose first patient by year-end with a systemically delivered CRISPR/Cas9-based therapy CAMBRIDGE, Mass., Oct. 19, 2020 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc…more
NEWS RELEASE 3-SEP-2019 Boston Medical Center and Columbia University Irving Medical Center awarded grant for new trial BOSTON – Researchers at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM), in collaboration with Columbia University Irving Medical Center (CUIMC), are le…more
Eidos Therapeutics Initiates ATTRibute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint
February 27, 2019 07:00 ET – Source: Eidos Therapeutics, Inc. San Francisco, Feb. 27, 2019 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced the initiation and design of its pivotal global Phase 3 trial (ATTRibute-CM) of AG10 in patients with transthyretin (TTR) amy…more
Alnylam Pharmaceuticals, Inc. announced today that the FDA has granted Orphan Drug Designation to ALN-TTRsc02. This drug has the potential to be a once-quarterly, low volume, subcutaneously administered RNAi medication in the management of ATTR amyloidosis. The FDA Orphan Drug Designation Program pr…more
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…more
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