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Rituximab (Rituxan®, Genentech, Inc., South San Francisco, CA, USA) is a chimeric anti-CD20 antibody that shows efficacy against B-cell lymphomas (James & Dubs, 1997) and has recently been approved as first line therapy in combination with chemotherapy for treatment of CD20+ NHLs (Cvetkovic & Perry, 2006).
Selective Laser Trabeculoplasty (SLT) has been advocated as a primary treatment for open-angle glaucoma 41 and there is evidence that shows efficacy of SLT as either an adjunct or an alternative to medical therapy where access to medications or compliance is a problem.
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In vivo studies are particularly called for as extracts that show efficacy in vitro will not necessarily lead to a corresponding response in vivo due to the dose used and associated metabolic responses.
Failed consecutive prophylactic treatment trials with at least three agents that showed efficacy over placebo in randomized controlled studies, used at the maximum tolerated dose over a sufficient period of time.
The use of stem cells in orthopedics has been researched for many years, with robust animal data that show efficacy in cartilage healing, tendon repair, and intervertebral disk treatment.
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Clinical trials have been performed using T cell gene transfer that show efficacy [9], but T cells have a limited life span in vivo which will likely limit the utility of this approach.
Chronic dietary administration of TBE-31 at doses that show efficacy is not toxic.
In addition, the FDA recently revised their guidelines for the development of therapeutics in SLE, permitting the approval of drugs that show efficacy in one organ system [ 6].
Omega-3 long-chain polyunsaturated fatty acids are safe diet supplements that showed efficacy in the prevention and therapy of cardiovascular diseases, dyslipidemia and metabolic syndrome [ 16- 19].
Many of these labs are already trying to find compounds that show efficacy in those models, in the hope that a pharmaceutical or biotech company will become interested in developing them further.
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CEO of Professional Science Editing for Scientists @ prosciediting.com