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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.
ISHLT issues new list of criteria to determine patient eligibility for heart transplant

ISHLT issues new list of criteria to determine patient eligibility for heart transplant

To determine patient eligibility for heart transplant, the International Society for Heart Lung Transplantation maintains a list of criteria, first issued in 2006, that acts as a guideline for physicians. [More]
FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

Ionis Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to IONIS-HTTRx for the treatment of patients with Huntington's disease (HD). [More]
Benjamin Wolozin receives $500,000 award in Alzheimer's Disease Drug Discovery Research

Benjamin Wolozin receives $500,000 award in Alzheimer's Disease Drug Discovery Research

Research that leads to improved therapies for Alzheimer's disease (AD) patients is one goal of Boston University School of Medicine professor of pharmacology and neurology Benjamin Wolozin, MD, PhD. He was one of six researchers awarded a two-year, $500,000 award in Alzheimer's Disease Drug Discovery Research by the Edward N. & Della L. Thome Memorial Foundation. [More]
Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Isis Pharmaceuticals, Inc. today announced that its partner, Biogen, has initiated a Phase 1/2 clinical study of ISIS-SOD1 Rx (BIIB067) in patients with amyotrophic lateral sclerosis (ALS). [More]
Takeda presents Phase 3 TOURMALINE-MM1 data for multiple myeloma at ASH 2015

Takeda presents Phase 3 TOURMALINE-MM1 data for multiple myeloma at ASH 2015

Takeda Pharmaceutical Company Limited today announced results from the TOURMALINE-MM1 trial presented at the 57th Annual Meeting and Exposition of the American Society of Hematology (ASH), showing that treatment with NINLARO (ixazomib) capsules is effective in extending progression free survival (PFS) with a manageable tolerability profile in patients with relapsed and/or refractory multiple myeloma. [More]
Study reveals how MGCs dispose waste that obstructs normal physiological processes

Study reveals how MGCs dispose waste that obstructs normal physiological processes

If rubbish is too big and unwieldy for normal household waste, its removal becomes the job of specialized experts. Researchers from the Technical University of Munich (TUM) have now discovered, in cooperation with colleagues from the UK, how large, fused cells help our body to deal with bulky items that may otherwise obstruct normal physiological processes. [More]
Brain ageing acceleration ‘nearly inevitable’ with age

Brain ageing acceleration ‘nearly inevitable’ with age

A large longitudinal study has charted the ages and rates at which people develop amyloidosis and neurodegeneration. [More]
Researchers find molecular mechanism that may be responsible for development of amyloidoses

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. [More]
Approval to start trial of Videregen's replacement trachea technology

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. [More]
Alnylam presents positive ongoing phase 2 open-label extension data for Patisiran and Revusiran

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). [More]
Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

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. [More]
Discovery may lead to development of cell-based regenerative therapy to restore thyroid function

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. [More]
OSU discovery sheds new light on how proteins fold

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. [More]
FDA awards research grants to boost product development for patients with rare diseases

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

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. [More]
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]
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