AL amyloidosis is a rare condition caused when bone marrow produces abnormal antibodies that can’t be broken down and are deposited in tissues, interfering with organ function. Survival for patients with AL amyloidosis is related to cardiac involvement, making cardiac biomarkers an accurate measure for staging for risk stratification. BMC (Boston Medical Center) researchers created …
A new staging system developed with a more accessible test to predict the chance of survival in patients living with light chain (AL) amyloidosis. Led by researchers from the Amyloidosis Center at Boston University School of Medicine (BUSM) and Boston Medical Center (BMC), the test incorporates cardiac biomarkers to accurately identify the involvement of the heart …
A UCL-led study has confirmed that some vials of a hormone used in discontinued medical treatments contained seeds of a protein implicated in Alzheimer’s disease, and are able to seed amyloid pathology in mice. The research, published in Nature, follows on from the team’s 2015 study that found evidence of amyloid pathology in people who …
Amyloid Pathology Transmission in Lab Mice and Historic Medical Treatments Read More »
Pfizer Inc. announced today that the US Food and Drug Administration (FDA) accepted for filing the company’s New Drug Applications (NDAs) for tafamidis for the treatment of Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Pfizer has submitted two NDAs based on two forms of Tafamidis: meglumine salt and free acid. Tafamidis is the only product to complete a …
Eidos Therapeutics, Inc., today announced the initiation and design of its pivotal global Phase 3 trial (ATTRibute-CM) of AG10 in patients with Transthyretin (TTR) Amyloid Cardiomyopathy (ATTR-CM). The design of the ATTRibute-CM study, which incorporates feedback from FDA, includes two potentially registrational endpoints. In Part A, benefit in change from baseline in 6-minute walk distance …
Amyloidosis is a rare condition that used to almost always be fatal, but doctors are finally making progress in treating it. “I had so many symptoms, and I really didn’t think they all connected,” Pam Rafferty said. Rafferty had a thick tongue, leg swelling, frothy urine, carpal tunnel syndrome, and then… “I started having heart …
Although most patients with familial amyloid polyneuropathy (FAP) who undergo liver transplant will require a pacemaker, implanting the device before the transplant is not beneficial, according to a new study. The research, “Pacemaker implantation in familial amyloid polyneuropathy: when and for whom?,” was published in the Journal of Interventional Cardiac Electrophysiology. Cardiac manifestations in patients …
Amyloidosis is a disorder in which certain proteins abnormally change their shape in a process called “misfolding.” The misfolded proteins accumulate together and form protein deposits called “amyloid fibrils” that may build up in your heart, kidneys or other organs. One of the main types of amyloidosis is called transthyretin (ATTR) amyloidosis. In this type, …
Why There’s New Hope if You Have Cardiac Amyloidosis Read More »
Scientists at Scripps Research have identified a group of small molecules that prevent structural changes to proteins that are at the root of AL amyloidosis, a progressive and often fatal disease. The small molecules bind to immunoglobulin proteins, which play an essential role in the body’s immune system, and then stabilize subunits of the protein …
Molecules That Curb Errant Proteins of AL Amyloidosis Point to New Type of Therapy Read More »
Akcea Therapeutics UK Ltd., has announced that NICE has issued a positive Final Evaluation Document (FED) for Tegsedi™ (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). This decision will allow patients in England with this rare, inherited, severely debilitating and fatal disease to access …
Tegsedi™ to be Available on the NHS for hATTR Amyloidosis Read More »