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FDA grants orphan designation for AMT's hemophilia gene therapy program

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B.

4 Jan 2012

First Edition: January 4, 2012

In today's headlines, Romney wins a historically close contest in Iowa caucuses and Wall Street Journal readers favor televising the Supreme Court arguments regarding the health law.

4 Jan 2012

Working in Kazakhstan to end HIV stigma, discrimination

In this post on USAID's "IMPACTblog," Erin McKee, USAID mission director for the Central Asian Republics, recounts a discussion roundtable with people "on the front lines" in the battle against HIV/AIDS in Kazakhstan.

4 Jan 2012

Researchers identify key ingredient needed for muscles to grow

We take it for granted, but the fact that our muscles grow when we work them makes them rather unique. Now, researchers have identified a key ingredient needed for that bulking up to take place. A factor produced in working muscle fibers apparently tells surrounding muscle stem cell "higher ups" that it's time to multiply and join in, according to a study in the January Cell Metabolism, a Cell Press journal.

4 Jan 2012

Task force identifies key themes and challenges in critical care research

To reduce mortality and improve patient care in the nation's ICUs, a task force formed by the Critical Care Societies Collaborative (CCSC), in conjunction with the US Critical Illness and Injury Trials Group (USCIITG) has recommended that research in the field become less fragmented and better account for patient heterogeneity and the complexity of critical illness.

4 Jan 2012

U.S. offers initial $125 million to UNHCR in 2012

"The United States said Thursday it will contribute an initial $125 million to the [U.N. High Commissioner for Refugees' (UNHCR)] 2012 operations, including support for refugees returning to Afghanistan and the Democratic Republic of Congo," Agence France-Presse reports.

4 Jan 2012

Yale study questions accuracy of relying on patient death rates to compare hospital quality

Hospitals, health insurers and patients often rely on patient death rates in hospitals to compare hospital quality. Now a new study by researchers at Yale School of Medicine questions the accuracy of that widely used approach and supports measuring patient deaths over a period of 30 days from admission even after they have left the hospital.

4 Jan 2012

One-third of NIH-funded trials remain unpublished 51 months after completion

In a study that investigates the challenges of disseminating clinical research findings in peer-reviewed biomedical journals, Yale School of Medicine researchers have found that fewer than half of a sample of trials primarily or partially funded by the National Institutes of Health (NIH) were published within 30 months of completing the clinical trial.

4 Jan 2012

Emerging forms of torture cause serious mental health problems

Use of torture around the world has not diminished but the techniques used have grown more complex and sophisticated, according to new research from Queen Mary, University of London.

4 Jan 2012

Chronic missed naps could put toddlers at risk for mood-related problems

A new study led by the University of Colorado Boulder could be a wake-up call for parents of toddlers: Daytime naps for your kids may be more important than you think.

4 Jan 2012

McConnell names conferees for tax-cut, doc fix negotiations

Late last month, Senate Minority Leader Mitch McConnell, R-Ky., named his picks to negotiate for a year-long extension of the payroll tax holiday and Medicare doc fix.

4 Jan 2012

PEDF may suppress brain from tumor-induced damage, metastasis

Scientists are one step closer to repairing the damage caused by brain metastasis, a major challenge in cancer treatment, according to data published in Cancer Research, a journal of the American Association for Cancer Research.

4 Jan 2012

Amyloid precursor protein fragment may be a biomarker for autism

Immune system abnormalities that mimic those seen with autism spectrum disorders have been linked to the amyloid precursor protein (APP), reports a research team from the University of South Florida's Department of Psychiatry and the Silver Child Development Center.

4 Jan 2012

Research reveals how bats parse duties at single neuron level

Imagine listening to music while carrying on a conversation with friends. This type of multi-tasking is fairly easy to do, right? That's because our brains efficiently and effectively separate the auditory signals - music to the right side; conversation to the left. But what researchers have not been able to do in humans or animals is to see a parsing of duties at the single neuron level - until now.

4 Jan 2012

Genomic architecture predisposes supercentenarians to age-related diseases

The first-ever published whole-genome sequences of not just one, but two supercentenarians, aged more than 114 years, reveal that both unusual and common genetic phenomena contribute to the genetic background of extreme human longevity.

4 Jan 2012

Study reveals possibility to selectively target and block crucial microRNA in liver cancer

A new study shows that it is possible to selectively target and block a particular microRNA that is important in liver cancer. The findings might offer a new therapy for this malignancy, which kills an estimated 549,000 people worldwide annually.

4 Jan 2012

JAMA highlights Oregon's Physician Orders for Life Sustaining Treatment program

Oregon's groundbreaking Physician Orders for Life Sustaining Treatment program (POLST) is featured in the latest edition of the Journal of The American Medical Association.

4 Jan 2012

CMS delays two anti-fraud projects

Modern Healthcare reports that the Centers for Medicare and Medicaid Services has put on hold its initiative targeting fraud related to power wheelchairs and its expansion of the Recovery Audit Contractors. Georgia Health News reports on how health care fraud has taken root in Georgia.

4 Jan 2012

MTR technique indicates that COPAXONE therapy results in tissue repair in RRMS patients

Researchers utilizing an advanced magnetic resonance imaging (MRI) technology to characterize and chart the evolution of MS lesions found that relapsing-remitting multiple sclerosis (RRMS) patients treated with COPAXONE (glatiramer acetate injection) experienced significantly increased magnetization transfer ratio.

4 Jan 2012

Losartan may help prevent smoking-related COPD

Working with mice, scientists at Johns Hopkins have successfully used a commonly prescribed blood pressure medicine, losartan (Cozaar), to prevent almost all of the lung damage caused from two months of exposure to cigarette smoke. The treatment specifically targeted lung tissue breakdown, airway wall thickening, inflammation and lung over-expansion.

4 Jan 2012