Darzalex Faspro is used to treat specific multiple myeloma patients and is also FDA-approved to treat adults with newly diagnosed light chain (AL) amyloidosis. Darzalex Faspro advantages over Darzalex are that it is faster to administer, it has less systemic administration-related reactions (ARRs)...
Chronologically (Fig.1), mRNA was first discovered by pioneering researches in 1961.4However, it was not until 1990 when Wolff et al. successfully expressed proteins by injecting mRNA into the body5that mRNA gradually gained recognition as a therapeutic modality.1In 1999, mRNA-engineered dendritic ...
Adjunctive treatment for patients with abnormal, viscid, or inspissated mucous secretions associated with conditions such as acute and chronic bronchopulmonary disorders (e.g., pneumonia, bronchitis, emphysema, tracheobronchitis, chronic asthmatic bronchitis, tuberculosis, bronchiectasis, primary amyloidosis ...
New Drugs Treat Rare Heart Disease Caused by Transthyretin-Mediated Amyloidosisdoi:info:doi/10.1097/01.NAJ.0000580244.26161.f0Aschenbrenner, Diane S.AJN
Hereditary transthyretin amyloidosis (hATTR amyloidosis) is a fatal autosomal dominant genetic condition caused by mutations in the gene encoding transthyretin (TTR), a liver protein involved in the transport of thyroxine and retinol63. The gene mutations cause TTR protein instability leading to its dis...
Low-molecular-weight heparins (LMWHs) are polypharmacologic drugs used to treat thrombotic and cardiovascular disorders. These drugs are manufactured using... W Jeske,J Walenga,D Hoppensteadt,... - 《Seminars in Thrombosis & Hemostasis》 被引量: 72发表: 2008年 The Quality Standards Subcommittee...
Interestingly, each approved orphan drug in the three of the four cases used different modalities; siRNA, antisense oligonucleotide and small molecule in transthyretin monomer reducer targeting transthyretin amyloidosis (ATTR), antisense oligonucleotide and small molecule in SMN2 mRNA splicing modifier ...
In 2006 Connolly et al. studied renal amyloidosis in intravenous drug users, but their work was limited due to the fact that it was not specified as to whether heroin or cocaine was used, and secondly only patients with amyloidosis were chosen for the study. 90% of the patients had ...
hereditary transthyretin-mediated amyloidosis by degrading mRNA encoding transthyretin (TTR).97Thus far, six siRNA drugs have received international approval. With the ongoing development of novel chemical modifications and targeted delivery systems, more siRNA drugs are anticipated to enter the market soon...
One is to transfer it into the patient's cells ex vivo; these transfected cells are then adoptively admin- istered back to the patient. This method is being investi- gated for genome engineering, genetic reprogramming, T cell- and dendritic cell (DC)‑based immunotherapies to treat ...