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[ Reversal Of Fragile X Syndrome In Mouse Model ]

Reversal Of Fragile X Syndrome In Mouse Model

A recent study finds that a new compound reverses many of the major symptoms associated with Fragile X syndrome (FXS), the most common form of inherited intellectual disability and a leading cause of autism. The paper, published by Cell Press in the April 12 issue of the journal Neuron, describes the exciting observation that the FXS correction can occur in adult mice, after the symptoms of the condition have already been established. Fragile X patients suffer from a complex set of neuropsychiatric symptoms of varying severity which include anxiety, hyperactivity, learning and memory deficits, low IQ, social and communication deficits, and seizures. Previous research has suggested that inhibition of mGlu5, a subtype of receptor for the excitatory neurotransmitter glutamate, may be useful for ameliorating many of the major symptoms of the disease.

Full Reports From Trials Should Be Public: Regulators Respond To Tamiflu Recommendations

The full clinical study reports that drugs that have been authorized for use in patients should be made publicly available in order to allow independent re-analysis of the benefits and risks of such drugs, according to leading international experts who base their assertions on their experience with Tamiflu (oseltamivir). Tamiflu is classed by the World Health Organization as an essential drug and many countries have stockpiled the anti-influenza drug at great expense to taxpayers. But a recent Cochrane review on Tamiflu has shown that even more than ten thousand pages of regulatory evidence were not sufficient to clarify major discrepancies regarding the effects and mode of action of the drug. Writing in this week's PLoS Medicine, Peter Doshi from Johns Hopkins University School of Medicine in Baltimore, USA, Tom Jefferson from the Cochrane Collaboration in Rome, Italy, and Chris Del Mar from Bond University in the Gold Coast, Australia say that there are strong ethical arguments for ensuring that all clinical study reports are publicly accessible.

Association Between Protein Aurora-A And Survival In Head And Neck Cancer

Researchers at Fox Chase Cancer Center in Philadelphia have found that a protein associated with other cancers appears to also be important in head and neck cancer, and may consequently serve as a good target for new treatments. The findings were reported at the AACR Annual Meeting. The researchers found that patients whose tumors had higher levels of the protein known as Aurora-A had a shorter survival following surgery to remove their tumors than patients whose tumors had normal levels of the protein. "This finding suggests Aurora A does play a role in the development of head and neck cancers, " says study author Christian J. Fidler, M.D., chief fellow in medical oncology at Fox Chase. "Consequently, Aurora-A represents another potential target for additional therapies." Previous research has associated Aurora-A with other cancer types, such as genitourinary, gastrointestinal, breast and lung cancers.

Roche's Trastuzumab Emtansine Has Positive Results Against Metastatic Breast Cancer

Roche released an announcement today in regards to its phase three trial called EMILIA. The program compared performance of its new drug Trastuzumab Emtansine against standard treatments for HER2-positive Metastatic Breast Cancer that use lapatinib plus Xeloda ® (capecitabine). Patients were enrolled in the study having previously received Herceptin ® and ataxane (chemotherapy). The trial the compared the patients on each treatment looking at progression-free survival, (PFS) ie. how long the patients lived without their disease becoming worse. Overall survival rates are not ready for release yet, but it appears that Trastuzumab Emtansine controlled the metastatic cancer better than standard treatments. Hal Barron, M.D., Chief Medical Officer and Head, Global Product Development explains that : "Trastuzumab emtansine represents a new approach for the treatment of patients with HER2-positive breast cancer that comes from our decades of research on the HER pathway .

Postmenopausal Osteoporosis Phase 3 Trial Starts, Say Amgen And UCB

Osteoporosis affects more than 75 million people, with women being four times more at risk of developing the disease than men. Osteoporosis is a chronic, progressive and systemic disease, whereby the bone tissue deteriorates, losing mass and strength, which makes the bones more fragile and increases the risk of fractures. Due to estrogen deficiency, the disease accelerates during and after women's menopause as ovarian function decreases during menopause, whilst the risk of fracture progressively increases with age. A phase 3 clinical trial program of the sclerostin antibody (CDP7851/AMG 785) for the treatment of postmenopausal osteoporosis (PMO) has just been announced by UCB and Amgen. CDP7851/AMG 785 is a humanized monoclonal antibody, which binds to sclerostin and inhibits it. Sclerostin is a protein secreted by bone cells, which inhibits bone formation.

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