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Toxic protein assemblies, or “amyloids,” long considered being key drivers in many neuromuscular diseases, also playing a beneficial role in the development of healthy muscle tissue, University of Colorado Boulder researchers have found. “Ours is the first study to show that amyloid-like structures not only exist in healthy skeletal muscle during regeneration, but are likely …

Amyloid Assemblies Once Believed to be Toxic Found to Play Key Role in Muscle Generation Read More »

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 …

New, More Accessible Staging System Developed to Predict Survival for Patients With AL Amyloidosis Read More »

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 …

New, More Accessible Staging System Developed to Predict Survival for Patients With Light Chain Amyloidosis Read More »

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 …

US FDA Accepts Regulatory Submissions for Review of Tafamidis to Treat Transthyretin Amyloid Cardiomyopathy Read More »

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 …

Eidos Therapeutics Initiates ATTRibute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint Read More »

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 …

Doctors Making Progress in Treating Amyloidosis Read More »

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 …

Implanting Pacemaker Before Liver Transplant Does Not Lead to Better Outcomes, Study Reveals 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 »