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EC approves Vertex's KALYDECO for treatment of cystic fibrosis

Vertex Pharmaceuticals Incorporated announced today that the European Commission has approved KALYDECOTM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

28 Jul 2012

PedBrain Tumor network publishes evaluation of first 125 genome analyses of medulloblastomas

Brain tumors are the primary cause of cancer mortality in children. Even if a cure is possible, young patients often suffer from the stressful treatment which can be harmful to the developing brain. The most common childhood brain tumors are medulloblastoma and pylocytic astrocytoma.

27 Jul 2012

Viral evolution and host protein levels predict rapid disease progression in hepatitis C patients

Scientists at the National Institutes of Health have identified several factors in people infected with the hepatitis C virus that may predict whether the unusually rapid progression of disease from initial infection to severe liver conditions, such as cirrhosis, will occur. Knowing whether a patient's condition is likely to deteriorate quickly could help physicians decide on the best course of treatment.

27 Jul 2012

Vaccine complex delivered safely by piggybacking onto 3D DNA nanostructures

In a quest to make safer and more effective vaccines, scientists at the Biodesign Institute- at Arizona State University have turned to a promising field called DNA nanotechnology to make an entirely new class of synthetic vaccines.

27 Jul 2012

Bone marrow transplant eradicates traces of HIV in men

Two men with longstanding HIV infections no longer have detectable HIV in their blood cells following bone marrow transplants. The virus was easily detected in blood lymphocytes of both men prior to their transplants but became undetectable by eight months post-transplant. The men, who were treated at Brigham and Women's Hospital (BWH), have remained on anti-retroviral therapy.

27 Jul 2012

Fusion of FGFR and TACC gene leads to glioblastoma

Researchers at Columbia University Medical Center (CUMC) have discovered that some cases of glioblastoma, the most common and aggressive form of primary brain cancer, are caused by the fusion of two adjacent genes.

26 Jul 2012

Illiteracy linked to schizophrenia risk

The risk for schizophrenia is significantly higher among illiterate than literate individuals in China, particularly among younger age groups, results from a population-based study show.

26 Jul 2012

Congenital long QT syndrome guidelines ‘too restrictive’

Athletes who choose to continue competing after being diagnosed with long QT syndrome have a low rate of LQTS-triggered cardiac events, US researchers report.

26 Jul 2012

Myotonia less important to patients with myotonic dystrophy than other symptoms

Complex, multi-system diseases like myotonic dystrophy - the most common adult form of muscular dystrophy - require physicians and patients to identify which symptoms impact quality of life and, consequently, what treatments should take priority. However, a new study out this month in the journal Neurology reveals that there is often a disconnect between the two groups over which symptoms are more important, a phenomenon that not only impacts care but also the direction of research into new therapies.

26 Jul 2012

Insomnia: an interview with Dr Darryle Schoepp

Insomnia is a very common disorder. In medical terms it can affect a third of people. It affects elderly as well as non-elderly people, and is more common in women.

26 Jul 2012

Rapamycin improves function and extends survival in mice with DCM and rare muscular dystrophies

Rapamycin, an immunosuppressant drug used in a variety of disease indications and under study in aging research labs around the world, improved function and extended survival in mice suffering from a genetic mutation which leads to dilated cardiomyopathy (DCM) and rare muscular dystrophies in humans.

26 Jul 2012

AAQ chemical temporarily restores vision in blind mice

A team of University of California, Berkeley, scientists in collaboration with researchers at the University of Munich and University of Washington in Seattle has discovered a chemical that temporarily restores some vision to blind mice, and is working on an improved compound that may someday allow people with degenerative blindness to see again.

26 Jul 2012

Researchers discover new cause of congenital myopathy

University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.

26 Jul 2012

Low blood anticoagulant levels increase risk for VTE

Low plasma levels of antithrombin, protein C, and protein S significantly increase the risk for venous thromboembolism, Italian research shows.

25 Jul 2012

ANCA-associated vasculitis not a single clinical entity

Study findings show distinct subtypes of antineutrophil cytoplasmic antibody-associated vasculitis, distinguished by the target of autoantigen reactivity.

25 Jul 2012

bluebird bio completes $60 million Series D financing

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.

25 Jul 2012

Alexion second quarter net product sales increase to $274.7 million

Alexion Pharmaceuticals, Inc. today announced financial results for the three and six months ended June 30, 2012.

25 Jul 2012

Rutgers cell biologist to receive Presidential Early Career Award for Scientists and Engineers

Fewer than 100 researchers nationwide are chosen each year to receive the Presidential Early Career Award for Scientists and Engineers (PECASE), and this year Dr. Nihal Altan-Bonnet is one of the 96 to receive this honor.

25 Jul 2012

Researchers discover bats as reservoir of infections

Measles, mumps, pneumonia, influenza and encephalitis in man, Carré's disease in dogs, Ovine Rinderpest (PPR)… all of these diseases are caused by viruses from the same family: Paramyxoviridae. A vast international study, carried out in collaboration with IRD researchers and published in Nature communications has led to the discovery of more than 60 new species of these dangerous infectious agents, almost double the number previously recorded. This family of highly diverse pathogens affects all animals, from canines to fowl, cattle and humans.

25 Jul 2012

Giving patients a new immune system can cure Crohn's disease

Researchers at Fred Hutchinson Cancer Research Center have opened a clinical trial to test the theory that giving a patient a new immune system can cure severe cases of Crohn's disease, a chronic inflammatory condition of the gastrointestinal tract.

25 Jul 2012