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Discovery Links Proteins Necessary To Repair Membranes
Researchers at UMDNJ-Robert Wood Johnson Medical School are a step closer to treating, and perhaps preventing, muscle damage caused by disease and aging. In their study, published in the June issue of Journal of Biological Chemistry, the scientists have linked the newly discovered protein MG53 to a pathway that repairs human muscle tissue along with the proteins caveolin-3 (Cav3) and dysferlin. Prior to this study, the underlying interactions that inhibited membrane repair in muscle tissue were unknown. Linking these proteins creates a mechanism that allows damaged membranes to be repaired, which may transform treatment for patients who suffer from severe complications of diseases such as muscular dystrophy, as well as cardiovascular disorders and conditions related to advancing age.
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Lexicon's Drug Candidate LX1032 For Carcinoid Syndrome Receives Orphan Drug Designation From EMEA
Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), a biopharmaceutical company focused on discovering and developing breakthrough treatments for human disease, announced today that it has received orphan drug designation from the Committee for Orphan Medical Products (COMP) of the European Medicines Agency (EMEA) for LX1032, the company"s oral drug candidate for managing gastrointestinal symptoms associated with carcinoid syndrome. LX1032 is designed to reduce serotonin production in patients with metastatic carcinoid tumors. Elevated levels of serotonin contribute to the gastrointestinal and possibly other symptoms experienced by these patients.
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Poniard Rally To Continue: Strong Efficacy And Safety Data From Picoplatin
Poniard announced positive Phase II data from its two trials in CRC and CRPC with picoplatin this morning, including efficacy that was comparable to the current standard of care, with a significantly improved safety profile. We believe that these data provide further evidence of picoplatin"s favorable efficacy and safety profile in two more large oncology markets, in addition to SCLC, and position picoplatin as a platform treatment that can provide solutions in multiple oncology settings. We believe that given these data from CRC and CRPC, in addition to the anticipated Phase III SPEAR data in SCLC, will attract the interest of multiple players and we expect that Poniard will be able to at least secure a large pharma partnership sometime this summer, unless a company like Sanofi (SNY Not Rated), Takeda (TKD Not rated), or Bristol-Myers Squibb (BMY Not Rated) decide to step in and acquire the whole company, for rights to both the IV and oral version of picoplatin.
Public Health

Cancer Risk In Delaying Sex Assignment Surgery

An editorial and a case report on Disorders of Sexual Development (DSD) in the Medical Journal of Australia highlight the need for early diagnosis and effective risk management in these rare but potentially life threatening cases. DSD describes conditions in which patients are born with ambiguous genitalia; DSD were previously described by terms such as "intersex", "hermaphroditism" and "testicular feminisation". In the editorial, endocrinologists from Melbourne"s Royal Children"s Hospital, Dr Garry Warne and Dr Jacqueline Hewitt, discuss the sometimes contentious management of the disorder, and call for the preparation of separate risk management strategies for individual patients. "International debate continues about the ethics of performing genital surgery on affected infants and children," Dr Warne said. "The option to assign a gender but postpone surgery until the child is able to give consent has been strongly advocated in some quarters, but hasn"t gained wide acceptance because of concerns that children might suffer psychological harm if left with ambiguous genitalia. "Another dilemma in delaying sex-assignment surgery is the increased risk of gonadal malignancy. what has largely been missing from the debate is recognition that surgery forms a necessary part of the risk management strategy for preventing gonadal malignancy." Gonadal malignancy proved fatal in a case detailed in the MJA by Dr Jim Parker (et al) from the University of Western Sydney. The case was of a 59 year old man who presented with previously undiagnosed DSD. Internal investigations for a related complaint revealed a normally formed uterus and an abdominal gonad with an advanced malignancy. The uterus and gonad were surgically removed, and the patient was treated postoperatively with chemotherapy, but died 18 months later. Dr Warne said the tragic case highlighted the need for accurate diagnosis and early monitoring and care from a multidisciplinary team. "The means for accurate diagnosis are improving with the development of microarray technology that offers an exciting and promising advance in identifying genetic mutations," Dr Warne said. Medical Journal of Australia


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