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Researchers develop molecule that binds to GAA enzymes key to progress of Pompe disease

Researchers develop molecule that binds to GAA enzymes key to progress of Pompe disease

Researchers at Leiden University have made a breakthrough in the study of the hereditary Pompe disease. Together with colleagues in York, they have developed a molecule that binds to the enzyme that is key to the progress of the disease. The findings have been published in ACS Central Science. [More]
Combination of sugar and polyphenols may prevent occurrence of neurodegenerative disease

Combination of sugar and polyphenols may prevent occurrence of neurodegenerative disease

Catherine Aaron and Gabrielle Beaudry were 17 when they knocked on the door of the laboratory of Alex Parker, a neuroscience researcher at the University of Montreal Hospital Research Centre. While students at Collège Jean-de-Brébeuf in Montreal, they were looking for a mentor for an after-school research project. Two and half years later, the results of this scientific adventure were published today in the Journal of Agricultural and Food Chemistry. [More]
Study explores two new antibodies believed to cause myasthenia gravis

Study explores two new antibodies believed to cause myasthenia gravis

A study of patients from across the nation with myasthenia gravis is helping determine the incidence of two new antibodies believed to cause the disease, and whether these patients need different treatment strategies. [More]

ResMed announces definitive agreement to acquire Brightree

ResMed, the world's leading tech-driven medical device company and innovator in sleep-disordered breathing and respiratory care, today announced a definitive agreement to acquire privately held Brightree, a leader in business management and clinical software applications for the post-acute care industry. [More]
Identifying and avoiding wasteful or unnecessary medical tests

Identifying and avoiding wasteful or unnecessary medical tests

Too many tests at the doctor's office could cost you more than just dollars. In addition to the huge hit to your wallet, there's also the potential harm of false positives, and just because a test has traditionally been done for a condition doesn't mean it's the best way to treat it. [More]
Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Although the vast majority of pediatric spine surgeries are safe, a handful of neuromuscular conditions seem to fuel the risk of cardiac arrest during such operations, according to research led by investigators at the Johns Hopkins Children's Center. [More]
U of T study provides hope for treating ALS and FTD

U of T study provides hope for treating ALS and FTD

It's the most common cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), but until now scientists weren't sure how a specific gene caused these devastating diseases. Now researchers from the University of Toronto are one step closer to solving this incredibly complex puzzle, offering hope for treatment. [More]
Researchers unlock structural details of key protein to treat neuromuscular disease

Researchers unlock structural details of key protein to treat neuromuscular disease

Researchers at Rice University and Baylor College of Medicine have unlocked the structural details of a protein seen as key to treating a neuromuscular disease. [More]
Novel wound closure technique may reduce complication rates for patients with scoliosis

Novel wound closure technique may reduce complication rates for patients with scoliosis

Patients with scoliosis who undergo surgery may be less likely to develop an infection or other complications after the procedure when a novel wound closure technique pioneered at NYU Langone Medical Center is utilized, according to new research. [More]
Chronic inflammatory demyelinating polyneuropathy commonly misdiagnosed

Chronic inflammatory demyelinating polyneuropathy commonly misdiagnosed

Half of patients referred to a specialist centre with a diagnosis of chronic inflammatory demyelinating polyneuropathy do not actually have the condition, report 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]
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]
Immune system linked to death of motor neurons in Amyotrophic lateral sclerosis

Immune system linked to death of motor neurons in Amyotrophic lateral sclerosis

A previously unknown link between the immune system and the death of motor neurons in Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, has been discovered by scientists at the CHUM Research Centre and the University of Montreal. The finding paves the way to a whole new approach for finding a drug that can cure or at least slow the progression of such neurodegenerative diseases as ALS, Alzheimer's, Parkinson's and Huntington's diseases. [More]
Mount Sinai researchers present landmark studies at AATS meeting

Mount Sinai researchers present landmark studies at AATS meeting

Icahn School of Medicine at Mount Sinai researchers presented several landmark studies at the 2015 American Association for Thoracic Surgery meeting in Seattle. [More]
TGen scientists discover the likely cause of rare type of muscle weakness in six children

TGen scientists discover the likely cause of rare type of muscle weakness in six children

Scientists at the Translational Genomics Research Institute (TGen), using state-of-the-art genetic technology, have discovered the likely cause of a child's rare type of severe muscle weakness. [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]
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