Isis Pharmaceuticals, Inc. announced the initiation of a Phase 1b/2a clinical study of ISIS-STAT3Rx in patients with advanced metastatic hepatocellular carcinoma (HCC).
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Despite decades of research, relatively little is known about the identity of RNA molecules that are transported as part of the molecular process underpinning learning and memory.
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An investigational treatment for an inherited form of Lou Gehrig's disease has passed an early phase clinical trial for safety, researchers at Washington University School of Medicine in St. Louis and Massachusetts General Hospital report.
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Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.
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Xenon Pharmaceuticals Inc. is pleased to announce that it has been awarded BIOTECanada's Gold Leaf Company of the Year Award for 2013.
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Santaris Pharma A/S, a privately held biopharmaceutical company focused on developing medicines targeted to disease-related mRNAs and microRNAs, today announced a worldwide strategic alliance with Bristol-Myers Squibb to discover and develop novel medicines using Santaris Pharma's proprietary Locked Nucleic Acid Drug Platform.
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Roche and Isis Pharmaceuticals, Inc today announced that they have formed an alliance to develop treatments for Huntington's disease based on Isis' antisense oligonucleotide technology.
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The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis - blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease - may be a first step towards a new era in the treatment of such disorders.
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Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 clinical study for ISIS-APOARx, an antisense drug targeting apolipoprotein(a) for the treatment of atherosclerosis.
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Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.
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The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.
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By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease.
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The vast majority - approximately 80 percent - of our DNA does not code for proteins, yet it gets transcribed into RNA. These RNA molecules are called non-coding and fulfill multiple tasks in the cell. Alongside a well-studied group of small RNAs, there is also a class of so-called long non-coding RNAs consisting of more than 200 nucleotides.
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Powerful antisense drugs that target disease-associated genes to block their expression can be used to treat a broad range of diseases. Though antisense therapy has been proven effective, challenges remain in ensuring that the drugs reach their intended targets.
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Genzyme, a Sanofi company, and Isis Pharmaceuticals Inc., today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for KYNAMROTM (mipomersen sodium) injection.
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Research led by Dr. Suresh Alahari, the Fred Brazda Professor of Biochemistry and Molecular Biology at LSU Health Sciences Center New Orleans and its Stanley S. Scott Cancer Center, details exactly how the Her2 cancer gene promotes the progression and spread of breast cancer cells.
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Scientists from the Florida campus of The Scripps Research Institute (TSRI) have developed a new approach to alter the function of RNA in living cells by designing molecules that recognize and disable RNA targets. As a proof of principle, in the new study the team designed a molecule that disabled the RNA causing myotonic dystrophy.
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A muscle relaxant called dantrolene used for treating malignant hyperthermia boosts the activity of therapies currently being developed to treat the genetic disorder Duchenne muscular dystrophy (DMD), show study findings.
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Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degeneration and, eventually, death.
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Biogen Idec and Isis Pharmaceuticals, Inc. today announced that they have entered into a global collaboration agreement under which the companies will discover and develop antisense drugs against three undisclosed targets to treat neurological or neuromuscular disorders.
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