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.

Brain ageing acceleration ‘nearly inevitable’ with age

A large longitudinal study has charted the ages and rates at which people develop amyloidosis and neurodegeneration.

23 Nov 2015

Researchers find molecular mechanism that may be responsible for development of amyloidoses

A team of local researchers has proposed a molecular mechanism that may be responsible for the development of life-threatening diseases called amyloidoses. The best known of such diseases is Alzheimer's disease (AD), but there are many others that are receiving increased scrutiny, in part because of mounting evidence linking them to atherosclerosis and aging.

13 Nov 2015

Approval to start trial of Videregen's replacement trachea technology

The INSPIRE consortium, led by Videregen Ltd, has received approval from the MHRA1 to start a UK clinical trial with its tissue engineered replacement trachea.

3 Nov 2015

Alnylam presents positive ongoing phase 2 open-label extension data for Patisiran and Revusiran

Alnylam Pharmaceuticals, Inc. has announced new results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis).

3 Nov 2015

Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study evaluating ISIS-FXIRx in patients with end-stage renal disease (ESRD) on hemodialysis. In May 2015, Isis entered into an exclusive license agreement with Bayer HealthCare (Bayer) to develop and commercialize ISIS-FXIRx for the prevention of clotting disorders.

30 Oct 2015

Discovery may lead to development of cell-based regenerative therapy to restore thyroid function

A discovery made by investigators from Beth Israel Deaconess Medical Center and the Boston University School of Medicine may help lead to the development of a cell-based regenerative therapy which could be used to restore thyroid function in patients with cancer who have had their thyroids surgically removed and children born with congenital hypothyroidism.

23 Oct 2015

OSU discovery sheds new light on how proteins fold

Biochemists at Oregon State University have made a fundamental discovery about protein structure that sheds new light on how proteins fold, which is one of the most basic processes of life.

19 Oct 2015

FDA awards research grants to boost product development for patients with rare diseases

The U.S. Food and Drug Administration today announced it has awarded 18 new research grants totaling more than $19 million to boost the development of products for patients with rare diseases, which affect the lives of nearly 30 million Americans.

11 Oct 2015

Light-chain amyloidosis patients treated with high-dose chemo and stem cell transplantation have long-term survival

Patients with Light-chain (AL) amyloidosis who are treated with high-dose chemotherapy (melphalan) and autologous (one's own) stem cell transplantation (HDM/SCT) have the greatest success for long-term survival.

9 Oct 2015

Amyloid-beta kinetics reveal link between aging and AD risk

Slowed amyloid-beta turnover may explain aging’s association with increased amyloidosis and Alzheimer’s disease, say researchers.

12 Aug 2015

Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

AstraZeneca and Isis Pharmaceuticals, Inc. today announced a strategic collaboration to discover and develop antisense therapies for cardiovascular, metabolic and renal diseases.

3 Aug 2015

NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen

Isis Pharmaceuticals, Inc., the leader in RNA-targeted therapeutics, and Akcea Therapeutics, its wholly owned subsidiary, announced today that The New England Journal of Medicine (NEJM) has published positive clinical results from a Phase 2 clinical study evaluating volanesorsen (formerly ISIS-APOCIII Rx) in patients with very high to severely high triglycerides.

30 Jul 2015

Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Isis Pharmaceuticals, Inc. announced today that it has earned a $2.15 million milestone payment from Biogen related to advancing the ongoing pivotal Phase 3 study (CHERISH) evaluating ISIS-SMN Rx in children with spinal muscular atrophy (SMA).

14 Jul 2015

Akcea Therapeutics obtains FDA Orphan Drug Designation for volanesorsen

Akcea Therapeutics, a wholly-owned subsidiary of Isis Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS).

6 Jul 2015

More support for amyloidosis as earliest AD biomarker

A longitudinal study of people with autosomal dominant Alzheimer’s disease shows that β-amyloid deposition begins well before neurodegeneration and cognitive decline.

2 Jul 2015

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on children with spinal muscular atrophy (SMA) who have completed the open-label, Phase 2 multiple-dose study of ISIS-SMN Rx and are continuing to receive treatment in an open-label extension (OLE) study.

22 Jun 2015

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMN Rx in infants with Type I spinal muscular atrophy (SMA).

11 Jun 2015

Stemedica receives FDA's IND approval for itMSC Phase IIa clinical trial to treat Alzheimer's disease

Stemedica Cell Technologies, Inc. received the FDA's investigational new drug (IND) approval for a United States-based, Phase IIa clinical study using its allogeneic stem-cell therapy to treat subjects with mild to moderate dementia due to Alzheimer's disease (AD), the sixth leading cause of death in the United States.

9 Jun 2015

Alnylam announces clinical data from patisiran Phase 2 OLE study for treatment of patients with FAP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today initial 12-month clinical data from its ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP).

24 Apr 2015

Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-ANGPTL3Rx. In this study, healthy volunteers treated with ISIS-ANGPTL3Rx achieved dose-dependent, statistically significant reductions in angiopoietin-like 3 (ANGPTL3) of up to 93 percent with a mean reduction of up to 84 percent from baseline (p<0.001).

23 Mar 2015