cardiac amyloidosistransthyretinheart failuremedicationtherapyLIVER-TRANSPLANTATIONMOLECULAR TWEEZERSP COMPONENTHEREDITARYINHIBITORSSupplemental Digital Content is Available in the Text. Transthyretincardiac amyloidosis is a rare disease that has gained significant attention in recent years because of misfolding of ...
Hereditary transthyretin amyloidosis with polyneuropathy is a rare, progressive disease that causes damage to the peripheral nerves because of the accumulation of transthyretin amyloid fibrils (Adams et al.2021).Eplontersenis an N-acetylgalactosamine-linked antisense oligonucleotide that inhibits transthyretin ...
a medication currently used to treat different types of leukemia, was effective in the treatment of heavily pre-treated MM and AL-amyloidosis patients, a disease associated with plasma cell dyscrasia, with mild manageable adverse events and no associated deaths. ...
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...
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 elucidate its...
In a pooled safety population of 898 patients with multiple myeloma (N=705) or light chain (AL) amyloidosis (N=193) who received DARZALEX FASPRO® as monotherapy or in combination, 9% of patients experienced a systemic a...
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 medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intraven...
ULTOMIRIS®(ravulizumab-cwvz) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an u...
A biosimilar is a biological therapy (hormones and vaccines are examples) that has no meaningful difference from one that’s already FDA-approved and on the market—think of it as a generic medication that pharmacists can swap for a name-brand drug, but that doesn’t require prior approval ...