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Some Black Women With Advanced Breast Cancer Opt Against Treatment, Study Finds
A new study has found that some black women with advanced breast cancer declined treatment with chemotherapy or radiation, though researchers did not know the reason why so many of the women opted against treatment, HealthDay/Las Vegas NOW reports. For the study, researchers examined records for 107 women with cases of advanced breast cancer that were reported at one inner-city hospital between 2000 and 2006. Eighty-seven percent of the women were black, and 29% of them had breast cancer tumors that did not respond well to new, targeted treatments. Of all women, 20.5% declined chemotherapy and 26.3% opted against radiation.Lead researcher Monica Rizzo, an assistant professor of surgery at the Division of Surgical Oncology at the Emory University School of Medicine, said the reason why the women declined treatment is not clear, adding, "We looked at marital status, as well as religious background, of those women, and unfortunately, we were not able to find any clear identifier." Researchers speculated that fear of the medical system, poverty and cultural differences might play a role. Rizzo"s group has started a community outreach program through which a nurse practitioner and social worker follow up with breast cancer patients (Reinberg, HealthDay/Las Vegas NOW, 5/22). An abstract of the study is available online.
Public Health

AMT Receives EMEA Orphan Drug Designation For Acute Intermittent Porphyria

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced that the European Medicines Agency has granted Orphan Drug Designation to AMT"s gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP). Orphan Drug Designation for AIP entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-021 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval. "We are proud to have received this Orphan Drug Designation for the treatment of acute intermittent porphyria," said Sander van Deventer, Chief Executive Officer of AMT. "This designation is an important step in the development of a treatment for this seriously debilitating and potentially lethal disease." About Acute Intermittent Porphyria Acute intermittent porphyria is a rare genetic disease in which mutations in the PBGD gene result in insufficient activity of a protein necessary for the synthesis of heme. This leads to an accumulation of toxic intermediate metabolites resulting in a wide variety of problems including acute, severe abdominal pains, psychiatric, neurologic illnesses, and muscular weakness. Long-term consequences may be irreversible nerve damage, liver cancer and kidney failure. Acute porphyric attacks can be life-threatening. AIP affects 1 per 10.000 people in the European Union. Currently available therapies do not prevent the symptoms and consequences of acute porphyric attacks. AMT has demonstrated that its product, AMT-021, results in normalization of the PBGD protein in an animal model of AIP. In this model, the therapy completely prevented the occurrence of attacks and significantly ameliorated the neuropathy that develops in untreated mice. AMT-021 is intended to provide long-term normalization of the PBGD protein in order to prevent acute porphyric attacks and their complications. About Amsterdam Molecular Therapeutics AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT"s proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially those that are caused by one faulty gene. Currently, AMT has a product pipeline with nine products at different stages of development. Certain statements in this press release are "forward-looking statements" including those that refer to management"s plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT"s business, including, but not limited to, the timely commencement and success of AMT"s clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT"s products, effectiveness of AMT"s marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT"s initial public offering on June 20, 2007, and AMT"s public announcements made from time to time. Amsterdam Molecular Therapeutics B.V


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