Amyloidosis News and Research

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Amyloidosis is the term applied to a group of conditions caused by deposits of an abnormal protein called amyloid. This rare but serious condition affects between 1 and 5 people in every 100,000 each year in the UK. The condition is mainly seen among the elderly.

Genetic variant mainly found in individuals of African ancestry increases odds of heart failure

A genetic variant in the gene transthyretin (TTR)- which is found in about 3 percent of individuals of African ancestry- is a more significant cause of heart failure than previously believed, according to a multi-institution study led by researchers at Penn Medicine.

10 Dec 2019

Study identifies genetic variation linked to heart failure risk in people of African, Latino ancestry

A genetic variation believed to increase risk for heart failure in people of African or Latino ancestry has been identified in a new study by researchers from the Icahn School of Medicine at Mount Sinai and Perelman School of Medicine at the University of Pennsylvania.

9 Dec 2019

Novel amyloid protein that induces amyloidosis discovered

A collaboration led by scientists at Tokyo University of Agriculture and Technology, Japan, has discovered a novel amyloid protein that induces amyloidosis in rats.

25 Oct 2019

Study aims to achieve earlier diagnosis of ATTR cardiac amyloidosis in minority populations

Researchers at Boston Medical Center and Boston University School of Medicine, in collaboration with Columbia University Irving Medical Center, are leading a national, multi-site study aimed to achieve earlier diagnosis of transthyretin cardiac amyloidosis.

4 Sep 2019

Researchers identify possible role of polyphosphate in dialysis-related amyloidosis

Long-term dialysis treatment is tough on the body in many ways, but one of the most serious complications is dialysis-related amyloidosis, a disease characterized by abnormal buildup of protein aggregates—called amyloid fibrils—in joints, tissues and organs.

8 Jul 2019

Study provides new insights into key beneficial function of small ancient protein

Cell boundaries are made of lipids. When cells are severely damaged, these lipids need to be rapidly removed to avoid toxicity and facilitate tissue healing. Researchers have discovered that a small ancient protein, Serum Amyloid A (SAA), plays a previously unknown key role in this vital process.

21 May 2019

Long-term antibiotic treatment slows growth of amyloid plaques in male mice but not in females

Researchers at The University of Chicago have demonstrated that the type of bacteria living in the gut can influence the development of Alzheimer's disease symptoms in mice.

17 May 2019

Renal infarction linked with acute kidney injury in patients with cardiac amyloidosis

Systemic amyloidosis is a major cause of renal injury, mostly due to direct kidney damage caused by deposits of abnormal protein called amyloid, in the kidney parenchyma.

16 May 2019

Scientists identify promising small molecules that curb errant proteins of AL amyloidosis

Scientists at Scripps Research have identified a group of small molecules that prevent structural changes to proteins that are at the root of AL amyloidosis, a progressive and often fatal disease.

12 Apr 2019

Researchers use experimental PET to detect abnormal tau in brains of living former NFL players

Using an experimental positron emission tomography scan, researchers have found elevated amounts of abnormal tau protein in brain regions players with cognitive, mood and behavior symptoms.

11 Apr 2019

Researchers create new accessible staging system to predict survival for patients with AL amyloidosis

A new staging system developed with a more accessible test to predict the chance of survival in patients living with light chain (AL) amyloidosis.

8 Nov 2018

Alnylam announces positive results from Phase 1/2 study of lumasiran for treating primary hyperoxaluria-1

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today updated positive results from its Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1.

4 Oct 2018

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that it has submitted a Clinical Trial Authorization application to the Medicines and Healthcare products Regulatory Agency to initiate a Phase 1/2 study of ALN-AAT02, an investigational RNAi therapeutic targeting alpha-1 antitrypsin for the treatment of AAT deficiency-associated liver disease.

3 Oct 2018

Alnylam reports further progress on broadening the therapeutic potential of RNAi

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today further progress on the Company's platform efforts in extrahepatic delivery of novel siRNA conjugates, including central nervous system (CNS) and ocular delivery in rat and non-human primates (NHPs).

3 Oct 2018

Newly discovered protein shown to cause amyloid-beta plaques in Alzheimer's

Scientists have discovered that a potassium channel called KCNB1 can trigger the formation of amyloid-beta plaques seen in Alzheimer's disease.

2 Oct 2018

Alnylam announces positive results from Phase 3 Study of givosiran for treating acute hepatic porphyria

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today positive topline results from the interim analysis of the ENVISION Phase 3 Study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria.

27 Sep 2018

EC grants marketing authorization for ONPATTRO (patisiran) in Europe

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the European Commission (EC) has granted marketing authorization for ONPATTRO (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

30 Aug 2018

FDA approves first targeted RNA-based treatment for rare, fatal genetic disease

The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

10 Aug 2018

Janssen’s Darzalex receives positive CHMP opinion for treating multiple myeloma patients

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has recommended broadening the existing marketing authorization for Darzalex® for use as frontline therapy.

28 Jul 2018

Alnylam’s patisiran receives positive opinion from CHMP for treating hATTR amyloidosis

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Committee for Medicinal Products for Human Use has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

27 Jul 2018