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The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world's first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).
MSN: European regulators back first gene therapy drug
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Biogen Idec and partner Elan had to pull their multiple sclerosis drug Tysabri after patients in clinical trials came down with a rare viral disorder that can lead to brain damage or death.
FORBES: Genentech's Next Act
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In a rare peek at the costs of clinical trials, Peto discloses how much the studies he is involved with cost.
FORBES: Magazine Article
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Jack Lief , Arena's chief executive, points out that the heart valve side effects were rare, and that a big clinical trial could settle the issue.
FORBES: Fat Chances
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And his efforts were backed up by clinical finishing and solid defence on the all too rare occasions when it mattered, most crucially from Leeds old boy Richie Mathers.
BBC: Leeds 6-30 Warrington
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Although the beneficial effects of high-potency statins have been well-characterized in clinical trials, these same trials have lacked the power to illuminate rare but potentially important adverse events.
FORBES: High Potency Statins Linked To Increased Risk For Acute Kidney Injury
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Clinical use of Cox-2 drugs in association with cancer is still relatively rare.
FORBES: Magazine Article
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Novartis is confronting such concerns head-on with large clinical trials that try to tease out potential side effects and will take the rare step of directly comparing Prexige, formerly known as Cox-189, to both Celebrex and Vioxx.
FORBES: Novartis' Potential Blockbuster Zelnorm Approved
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The Rare and Imported Pathogens Laboratory at Porton Down, is providing diagnostic support to clinical teams to help them handle suspected anthrax cases.
BBC: Drugs
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Because the disease is so rare, the drug was tested in only 56 patients in its late-stage clinical trials.
FORBES: Pfizer Enters Market For Super-Expensive, Ultra-Rare Disease Drugs -- With A Price Cut
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It was an analysis of published clinical trials that raised the first big questions about drug-coated stents causing heart attacks in rare cases.
FORBES: Open-Source Drug Safety?