In recent years, the development of new drugs has brought new hope to patients. This review presents the latest advances in drug development and clinical application to provide a reference for clinicians managing transthyretin cardiac amyloidosis....
Amvuttra (vutrisiran) is an RNAi therapeutic for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis and cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis. Alnylam Announces FDA Approval of Amvuttra (vutrisiran), the First RNAi Therapeutic to Reduce...
Janssen has submitted a new combination regimen for its blockbuster multiple myeloma drug Darzalex for approval with the FDA and EMA, hoping to give the treatment another edge over emerging competitors
Ixazomib is a novel oral proteasome inhibitor which is being studied in multiple myeloma and systemic light-chain (AL) amyloidosis, and other malignancies. Ixazomib was granted orphan drug designation for the treatment of patients with relapsed or refractory multiple myeloma by the Ministry of Health,...
(Dhillon2023b; Rais et al.2023). Elranatamab has been approved for relapsed or refractory multiple myeloma after four or more prior therapies following priority review, breakthrough, and orphan drug designation (Dhillon2023b). Elranatamab is well tolerated, and the most common non-hematologic ...
This lipid nanoparticle (LNP)-encapsulated small interfering RNA (siRNA) was approved by the United States Food and Drug Administration (FDA) for the treatment of hereditary ATTR amyloidosis, marking a significant milestone in the field and opening the door for mRNA-based drugs to be used in ...
A trial run by Intellia Therapeutics and biotech company Regeneron treated the life-threatening disease transthyretin amyloidosis. Billions of microscopic structures carrying genetic instructions for the CRISPR gene-editor were administered by one-time intravenous injection. The lipid nanoparticles each carried...
Overall, metal dyshomeostasis is closely linked to various events in AD such as amyloidosis, tauopathy, oxidative stress, and neuronal death. This hypothesis provides an alternative approach to understanding the pathogenesis of AD and detecting pathological changes. Further research is necessary to ...
CRISPR infusion was used for the first time directly to edit human genes. Intravenous gene therapy marks a watershed in modern medicine. A trial run by Intellia Therapeutics and biotech company Regeneron treated the life-threatening diseasetransthyretin amyloidosis. Billions of microscopic structures carr...
It is provided a catechol-O-methyltransferase (COMT) inhibitor for use in the prevention and/or treatment of trans-thyretin-associated amyloidosis. It is also provided a catechol-O-