New treatments for amyloidosisAn anti-CD38 monoclonal antibody and an in vivo gene-editing approach show promise for the treatment of different forms of systemic amyloidosis in separate clinical trials.doi:10.1038/s41584-021-00680-5Sarah OnuoraNature Reviews Rheumatology...
This is an exciting time for the AL amyloidosis community as patient advocacy groups, academia, pharmaceutical companies, and regulatory agencies are joining in an effort to develop safe and effective treatments for patients with AL amyloidosis. Clinical research efforts in AL amyloidosis have focused ...
The evolution of blood tests that accurately predict brain amyloidosis and other AD pathology may greatly improve the feasibility of creating more homogeneous trial populations for assessment of symptomatic agents [113,114,115]. Precision drug development depends on demonstrating a pharmacodynamic effect ...
The Aβ hypothesis is the mainstream hypothesis for the pathogenesis of AD and has influenced the direction of drug development over the past three decades. According to the hypothesis, the pathological progression of AD originates from asymptomatic cerebral amyloidosis many years before the onset of...
(Engelberts et al.2020). Epcoritamab has been approved for relapsed or refractory diffuse large B cell lymphoma and high-grade B cell lymphoma after at least two prior systemic treatments via priority review and accelerated approval (Frampton2023). The most common treatment-related AE include ...
Cardio-Oncology and Heart Failure: AL Amyloidosis for the Heart Failure Clinician A Supplement to the Scientific Statement from the Heart Failure Society of AmericaDOI:10.1016/j.cardfail.2024.08.046 Heart Failure in Patients with Cancer – A Patient's Perspecti...
It is important to note that ferroptosis has also been closely linked to resistance to various cancer treatments. For example, several studies found that cancer cells with a high-mesenchymal state are more resistant to a variety of cancer treatments, but are particularly susceptible to ferroptosis-...
s pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including AL amyloidosis, ATTR amyloidosis, Alzheimer’s disease, Parkinson’s disease and a number of other neurodegenerative diseases. For more information, plea...
amyloidosis (hATTR).294,295,296Indeed, the rapid development of siRNA is benefit from lipid nanoparticles (LNPs) technology progress and related nucleic acid modification methods.297,298,299Researchers also use siRNAs for the treatment of autoimmune diseases and achieved some progress.300,301...
for the treatment of Sickle Cell Disease [26]. Additionally, CRISPR-Cas9 is being evaluated for in vivo delivery using mRNA-LNP system, with treating transthyretin amyloidosis initiated in 2021 [27]. In Fig.1A, mRNA technology is contextualized among other innovative therapeutic modalities for ...