Syndrome News and Research

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Methylation provides signal of unique mix of leukocytes

When a person is sick, there is a tell-tale sign in their blood: a different mix of the various types of immune cells called leukocytes. A group of scientists at several institutions including Brown University has discovered a way to determine that mix from the DNA in archival or fresh blood samples, potentially providing a practical new technology not only for medical research but also for clinical diagnosis and treatment monitoring of ailments including some cancers.

10 Jul 2012

Inspiration receives FDA clinical hold order for IB1001 phase III studies on hemophilia B

Ipsen announced today that its partner Inspiration Biopharmaceuticals Inc. (Inspiration) was notified by the Food and Drug Administration (FDA) that both clinical trials evaluating the safety and efficacy of IB1001, an investigational intravenous recombinant factor IX (rFIX) therapy for the treatment and prevention of bleeding episodes in people with hemophilia B, were placed on clinical hold.

10 Jul 2012

Gene defect linked to cluster of systemic complications

Research teams from The University of Texas Health Science Center at Houston (UTHealth) and Paris, France have discovered a gene defect linked to a cluster of systemic complications, including life-threatening thoracic aortic disease and intracranial aneurysms. The new syndrome is similar, but distinct from known syndromes such as Marfan and Loeys-Dietz syndrome.

9 Jul 2012

Regnite to treat primary restless legs syndrome launched in Japan

Astellas Pharma Inc. and XenoPort, Inc. announced today that Regnite (gabapentin enacarbil) Extended-Release Tablets will be launched in Japan on July 10, 2012. Regnite is approved in Japan for the treatment of moderate-to-severe primary restless legs syndrome (RLS).

9 Jul 2012

JRD receives FDA priority review designation for XARELTO sNDAs to treat PE, DVT

Janssen Research & Development, LLC (Janssen R&D) announced today the U.S. Food and Drug Administration (FDA) has assigned a priority review designation to the supplemental New Drug Applications (sNDAs) filed on May 2, 2012 for XARELTO (rivaroxaban), an oral anticoagulant, seeking new indications to treat patients with deep vein thrombosis (DVT), pulmonary embolism (PE), and to prevent recurrent DVT and PE.

9 Jul 2012

Does alcohol kill brain cells?

Several origins have been suggested for the idea that alcohol kills brain cells. Some link it to the temperance movement, which called for prohibition of alcohol.

9 Jul 2012

Health officials working to identify respiratory illness responsible for deaths of more than 60 Cambodian children

WHO and Cambodian health officials are investigating "the source of an unidentified illness" -- characterized by high fevers, severe respiratory problems, "and in some cases neurological symptoms" -- that has killed more than 60 children in the country since April, NPR's health blog "Shots" reports (Hensley, 7/5). "

7 Jul 2012

Protein AMPK protects sensory cells in the inner ear from permanent damage

If you're concerned about losing your hearing because of noise exposure (earbud deafness syndrome), a new discovery published online in the FASEB Journal offers some hope. That's because scientists from Germany and Canada show that the protein, AMPK, which protects cells during a lack of energy, also activates a channel protein in the cell membrane that allows potassium to leave the cell.

7 Jul 2012

New Gateway-funded phase II ADAPT study for metastatic colorectal cancer

The Gateway for Cancer Research announces an exciting new Gateway-funded phase II study using ADAPT therapy for treatment of metastatic colorectal cancer based on a 2010 AACR presentation by Dr. Lin and his collaborators.

7 Jul 2012

FDC precursor cells exist in the wall of blood vessels

Chronic inflammatory conditions are extremely common diseases in humans and in the entire animal kingdom. Both in autoimmune diseases and pathogen-caused diseases, the inflamed areas are rapidly colonized by antibody producing B lymphocytes - which organize themselves in highly structured areas called "lymphoid follicles".

6 Jul 2012

PHF21A gene plays a big role in Potocki-Shaffer syndrome

A gene whose mutation results in malformed faces and skulls as well as mental retardation has been found by scientists.

6 Jul 2012

Insight into understanding IDH mediated disease

Agios Pharmaceuticals, the leading biopharmaceutical company focused on discovering and developing novel drugs in cancer metabolism, announced today the publication of a new study that provides further evidence of the link between a mutated metabolic enzyme and acute myeloid leukemia (AML), one of the most common types of leukemia in adults.

6 Jul 2012

Researchers use maternal blood sample to sequence genome of an unborn baby

Researchers at the Stanford University School of Medicine have for the first time sequenced the genome of an unborn baby using only a blood sample from the mother.

5 Jul 2012

Scientists identify causative link between oncometabolite and onset of AML

A team of international scientists led by principal investigator Dr. Tak Mak at the Princess Margaret Cancer Centre, University Health Network, has identified a causative link between the product of a mutated metabolic enzyme and the onset of acute myeloid leukemia (AML), one of the most common types of leukemia in adults.

5 Jul 2012