Coffee Symposium for Doctors by Akcea - Multidisciplinary care for Hereditary ATTR Amyloidosis
: A 360 Degree Perspective
is an autosomal dominant genetic disease, which means every first-degree relative of an affected patient has a 50 percent chance of having the same gene mutation.
While acknowledging that the root cause of Alzheimer's are unknown, and probably multifactorial, the paper hypothesized a pathophysiologic time line beginning with three-stage preclinical phase: Stage 1: Asymptomatic cerebral amyloidosis
: amyloid positive PET brain imaging with an amyloid-binding ligand and/or a cerebrospinal fluid assay with low-amyloid-beta 42 in the presence of normal cognition.
is a rare disease that occurs when a substance called amyloid builds up in a person's organs.
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis
. The antibody is designed to bind to insoluble light chain amyloid protein, including both kappa and lambda subtypes.
The breast amyloidosis
was first described in 1973 and is extremely rare (6).
A case series and two trials have suggested that I L1-blocking therapy is efficient in preventing FMF attacks and progression of FMF-associated amyloidosis
He was subsequently diagnosed with and began treatment for systemic amyloidosis
Primary localized amyloidosis
of the ureter is a rare disorder that is clinically and radiographically difficult to distinguish from urothelial carcinoma.
Hereditary ATTR (hATTR) amyloidosis
is an inherited, progressive disease caused by a genetic mutation that results in the misfolding of transthyretin (TTR) proteins.
All the findings were highly suggestive of primary bladder amyloidosis
. To exclude systemic amyloidosis
, a further evaluation was performed.
THURSDAY, July 5, 2018 (HealthDay News) -- For patients with hereditary transthyretin amyloidosis
with polyneuropathy, an investigational RNA interference therapeutic agent (patisiran) and a 2'-O-methoxyethyl-modified antisense oligonucleotide (inotersen), which inhibits hepatic production of transthyretin, improve clinical manifestations of disease, according to two studies published in the July 5 issue of the New England Journal of Medicine.