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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.
Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA). [More]
Gene therapy transforms life for men with severe form of hemophilia B

Gene therapy transforms life for men with severe form of hemophilia B

Gene therapy developed at St. Jude Children's Research Hospital, University College London and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported. [More]
Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec related to the initiation of investigational new drug (IND)-supporting studies of ISIS-BIIB3Rx. ISIS-BIIB3Rx is a recently identified development candidate designed to inhibit an undisclosed target to treat a neurodegenerative disease. [More]
Isis Pharmaceuticals secures $18 million from GSK to advance ISIS-TTR Rx Phase 2/3 study

Isis Pharmaceuticals secures $18 million from GSK to advance ISIS-TTR Rx Phase 2/3 study

Isis Pharmaceuticals, Inc. announced today that it has earned an $18 million milestone payment from GlaxoSmithKline related to the advancement of the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). [More]
HICCC receives $18 million grant from the National Cancer Institute

HICCC receives $18 million grant from the National Cancer Institute

Outstanding basic research, a growing focus on translating discoveries into treatments, and a dedication to patient care have earned the Herbert Irving Comprehensive Cancer Center (HICCC) of Columbia University Medical Center (CUMC) and NewYork-Presbyterian Hospital an $18 million, five-year Cancer Center Support Grant from the National Cancer Institute (NCI). [More]
Dynamic nuclear polarization: an interview with Professor Robert Griffin, Massachusetts Institute of Technology

Dynamic nuclear polarization: an interview with Professor Robert Griffin, Massachusetts Institute of Technology

DNP, or dynamic nuclear polarization, is an NMR technique which transfers polarization from the electron spins onto the nuclear spins, using constant microwave irradiation to enable the transfer. [More]
Isis earns $1 million payment from GSK related to advancement of ISIS-TTRRx

Isis earns $1 million payment from GSK related to advancement of ISIS-TTRRx

Isis Pharmaceuticals, Inc. announced today that it has earned a $1 million milestone payment from GlaxoSmithKline (GSK) related to the advancement of the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). [More]
Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases. [More]
Research findings could lead to new approach to Alzheimer’s prevention

Research findings could lead to new approach to Alzheimer’s prevention

In surprise findings, scientists at The Scripps Research Institute have discovered that a protein with a propensity to form harmful aggregates in the body when produced in the liver protects against Alzheimer’s disease aggregates when it is produced in the brain. The results suggest that drugs that can boost the protein’s production specifically in neurons could one day help ward off Alzheimer’s disease. [More]
Drugs that boost protein's production in neurons could help ward off Alzheimer's disease

Drugs that boost protein's production in neurons could help ward off Alzheimer's disease

In surprise findings, scientists at The Scripps Research Institute (TSRI) have discovered that a protein with a propensity to form harmful aggregates in the body when produced in the liver protects against Alzheimer's disease aggregates when it is produced in the brain. [More]
Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals, Inc. today reported a pro forma net operating loss (NOL) of $22.6 million for the three months ended March 31, 2014 compared to pro forma operating income of $4.5 million for the same period in 2013. [More]
Royal Free London NHS Foundation Trust selects OpenText to manage scanned legacy case-notes

Royal Free London NHS Foundation Trust selects OpenText to manage scanned legacy case-notes

OpenText, the global leader in Enterprise Information Management (EIM), announced today that it has been selected by the Royal Free London NHS Foundation Trust to manage its scanned legacy case-notes. [More]
RNA interference approach holds great promise for treating cancer and other diseases

RNA interference approach holds great promise for treating cancer and other diseases

Inspired by tiny particles that carry cholesterol through the body, MIT chemical engineers have designed nanoparticles that can deliver snippets of genetic material that turn off disease-causing genes. [More]
Early onset of dietary treatment may slow down progression of Alzheimer's disease

Early onset of dietary treatment may slow down progression of Alzheimer's disease

New research findings indicate that an early onset of dietary treatment may slow down the progression of Alzheimer's disease. The study was conducted on mice, and the results will be published in the February issue of Journal of Nutritional Biochemistry. Researchers from the University of Eastern Finland played a key role in the study, which was carried out as part of the LiPiDiDiet project funded by the European Union. [More]
Early onset of dietary treatment may slow down progression of Alzheimer's, says research

Early onset of dietary treatment may slow down progression of Alzheimer's, says research

New research findings indicate that an early onset of dietary treatment may slow down the progression of Alzheimer's disease. The study was conducted on mice, and the results will be published in the February issue of Journal of Nutritional Biochemistry. [More]
Study: Cholesterol levels linked to early signs of Alzheimer's disease in the brain

Study: Cholesterol levels linked to early signs of Alzheimer's disease in the brain

High levels of "good" cholesterol and low levels of "bad" cholesterol are correlated with lower levels of the amyloid plaque deposition in the brain that is a hallmark of Alzheimer's disease, in a pattern that mirrors the relationship between good and bad cholesterol in cardiovascular disease, UC Davis researchers have found. [More]
Diflunisal drug reduces neurological decline in patients with familial transthyretin amyloidosis

Diflunisal drug reduces neurological decline in patients with familial transthyretin amyloidosis

A recent study led by researchers from the Amyloidosis Center at Boston University School of Medicine and Boston Medical Center demonstrates that diflunisal, a generic anti-inflammatory drug, successfully reduced neurological decline and preserved the quality of life in patients with familial transthyretin amyloidosis. Diflunisal is an inexpensive and safe medication marketed over the past 40 years for arthritis and pain. [More]
Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated a pilot Phase II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene in development for the treatment of TTR-mediated amyloidosis. [More]
Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans. [More]
Takeda presents clinical study results of oral proteasome inhibitor MLN9708 in multiple myeloma patients

Takeda presents clinical study results of oral proteasome inhibitor MLN9708 in multiple myeloma patients

Takeda Pharmaceutical Company Limited today announced final Phase 1 and preliminary Phase 2 results of a study combining oral investigational MLN9708 administered twice a week with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma. [More]