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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.
Amyloid-beta kinetics reveal link between aging and AD risk

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. [More]
Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

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. [More]
NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen

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

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). [More]
Akcea Therapeutics obtains FDA Orphan Drug Designation for volanesorsen

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). [More]
More support for amyloidosis as earliest AD biomarker

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

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

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). [More]
Stemedica receives FDA's IND approval for itMSC Phase IIa clinical trial to treat Alzheimer's disease

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. [More]

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). [More]
Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

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). [More]
Royal Free London NHS Foundation Trust deploys OpenText to manage patient medical care records

Royal Free London NHS Foundation Trust deploys OpenText to manage patient medical care records

OpenText, the global leader in Enterprise Information Management (EIM), today announced that the Royal Free London NHS Foundation Trust has implemented OpenText Content Suite to store and manage digitised copies of patient medical care records. [More]
Simple blood test could be developed to diagnose Alzheimer's disease, say UCLA researchers

Simple blood test could be developed to diagnose Alzheimer's disease, say UCLA researchers

UCLA researchers have provided the first evidence that a simple blood test could be developed to confirm the presence of beta amyloid proteins in the brain, which is a hallmark of Alzheimer's disease. [More]
Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-PKKRx. In this study, healthy volunteers treated with ISIS-PKKRx achieved dose-dependent reductions of up to 95 percent in prekallikrein, or PKK. ISIS-PKKRx is a RNA-targeted antisense drug designed to inhibit the production of PKK for the prophylactic treatment of hereditary angioedema (HAE). [More]
Study shows amyloid formation may link type 2 diabetes and Alzheimer disease

Study shows amyloid formation may link type 2 diabetes and Alzheimer disease

The pathological process amyloidosis, in which misfolded proteins (amyloids) form insoluble fibril deposits, occurs in many diseases, including Alzheimer disease (AD) and type 2 diabetes mellitus (T2D). [More]
Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis Pharmaceuticals, Inc. announced today it has earned a $5 million milestone payment from Biogen Idec associated with the validation of an undisclosed target to treat a neurological disorder under its broad strategic collaboration with Biogen Idec to develop drugs to treat patients with neurological disorders. [More]
Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec for initiating the investigational new drug (IND)-supporting studies of ISIS-BIIB4 Rx. [More]
Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

Isis Pharmaceuticals, Inc. announced today top-line results from a Phase 2 study of ISIS-PTP1B Rx in patients with type 2 diabetes. In the Phase 2 study patients treated with ISIS-PTP1B Rx achieved statistically significant reductions in body weight and hemoglobin A1c (HbA1c). [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA. [More]

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Stanley T. Crooke, Ph.D., M.D., founder, CEO and chairman of the board of directors of Isis Pharmaceuticals, Inc., has been awarded the 2014 SCRIP Lifetime Achievement Award, one of the most prestigious of its kind in the pharmaceutical and biotechnology industries worldwide. [More]
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