News & Meetings

Immix Biopharma Announces 100% Overall Response Rate (n=10); 23.7 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2023 Immix Biopharma, Inc. (Nasdaq:IMMX) 100% (10/10) overall response rate (ORR) and 70% (7/10) complete response (CR) rate observed in standard of care (Dara-CyBorD) relapsed/refractory AL Amyloidosis patients with median 6 …

Immix Biopharma Announces 100% Overall Response Rate (n=10); 23.7 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2023 Read More »

Updated research regarding the ANDROMEDA study presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois, indicated the efficacy of subcutaneous daratumumab (DARA SC) and cyclophosphamide, bortezomib, and dexamethasone (CyBorD) as a combination therapy in newly diagnosed light chain (AL) amyloidosis patients. Daratumumab is a previously approved CD38-directed cytolytic antibody …

Combination Therapy Holds Promise for Newly Diagnosed Systemic AL Amyloidosis Patients Read More »

A new hereditary ATTR (hATTR) amyloidosis treatment, inotersen, an investigational antisense drug, may not be far from a US Food and Drug Administration (FDA) approval. Positive phase 3 data released this past March displayed an improvement in quality of life by 50% in patients with the disease, and with no effective treatment available, a lot …

Inotersen: An Exciting Prospect for hATTR Amyloidosis Treatment Read More »

Once used to diagnose myocardial infarction, technetium-99m pyrophosphate (Tc 99m PYP) imaging may be reborn as an alternative to biopsy for diagnosing cardiac transthyretin (ATTR) amyloidosis in some patients. Interest in the test is growing as new therapies show promise in late-stage clinical trials, suggesting possibilities for treating the under-recognized condition. Clinicians have known about …

Resurrecting Tc 99m PYP: As Cardiac Amyloidosis Therapies Emerge, an Old Test Gains a New Purpose Read More »

The FDA today granted Alnylam Pharmaceuticals approval for a first-in-class small interfering ribonucleic acid (siRNA) treatment—the first therapy indicated for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults. Onpattro™ (patisiran) is an infusion treatment whose approval comes 20 years after the discovery of RNA interference. Onpattro is designed to interfere with RNA production of …

FDA Approves Alnylam’s Onpattro for Polyneuropathy of hATTR Amyloidosis Read More »

The FDA announced that it has approved patisiran infusion for the treatment of patients with peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis. The treatment (Onpattro, Alnylam Pharmaceuticals) is the first of its kind to be approved by the FDA for patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis, and the first FDA approval for …

FDA Approves Patisiran Infusion for Hereditary Transthyretin-Mediated Amyloidosis Read More »

Treatment with the investigational drug tafamidis was associated with a reduction in all-cause mortality and cardiovascular hospitalizations in patients with transthyretin (TTR) amyloid cardiomyopathy, and improved functional capacity and quality of life, the phase III ATTR-ACT trial found. The mortality rate at 30 months was 29.5% in the tafamidis-treated group compared with 42.9% in the …

ESC: Tafamidis Cuts Deaths, Admissions in TTR Cardiac Amyloidosis Read More »

Tiny protein structures called amyloids are key to understanding certain devastating age-related diseases. Aggregates, or sticky clumped-up amyloids, form plaques in the brain, and are the main culprits in the progression of Alzheimer’s and Huntington’s diseases. Amyloids are so tiny that they can’t be visualized using conventional microscopic techniques. A team of engineers at Washington …

Blink’ and You Won’t Miss Amyloids: Engineering Team Just Found New Way to See Proteins That Cause Alzheimer’s, Other Diseases Read More »

The US Food and Drug Administration (FDA) has approved inotersen (Tegsedi, Akcea Therapeutics and Ionis Pharmaceuticals) for the treatment of polyneuropathy (PN) in adults with hereditary transthyretin amyloidosis (hATTR). hATTR-PN is a rare, progressive condition in which a mutation in the transthyretin (TTR) gene can lead to TTR amyloidosis, a severe, progressive deposition of amyloid …

FDA OKs Inotersen (Tegsedi) for Hereditary ATTR With Polyneuropathy Read More »

Cardiac amyloidosis, traditionally considered a rare and dire disease, is receiving increasing attention as sophisticated diagnostics are identifying more patients and novel therapies are emerging to combat it. Among the latest signs of progress are the encouraging results of the ATTR-ACT study of the drug tafamidis presented as a late-breaking trial at the European Society …

Major Advances are Afoot in Management of ATTR Cardiac Amyloidosis Read More »