Raptor Pharmaceuticals and DaVita Clinical Research Launch Screening Program to Identify Patients With Late Onset Nephropathic Cystinosis


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Raptor PharmaceuticalCorp. (Nasdaq: RPTP) and DaVita Clinical Research(R) (DCR(R) ) (NYSE: DVA)today announced a collaboration to screen blood samples from patients withend-stage renal disease (ESRD) in an effort to identify patients withunrecognized late-onset nephropathic cystinosis. DCR will supply blood samples with clinical data annotation from DCR'sbiorepository of over four thousand patients with ESRD. The screening effortwill employ high-throughput genetic sequencing of the cystinosin, lysosomalcystine transporter (CTNS) gene, mutations of which result in cystinosis.Results of the collaboration may reveal new insights into the prevalence ofmissed late-onset cystinosis in this at-risk patient population. "Today, most nephropathic cystinosis patients are identified in infancy bypediatric nephrologists, yet, as with many genetic diseases, later-onsetpatients have been identified where the clinical manifestations ofcystinosis may go unrecognized well into adulthood," noted Christopher M.Starr, Ph.D., Raptor's chief executive officer. "We believe these late-onsetpatients are likely to present with ESRD as adults and this screeningprogram, initiated with this collaboration, may help adult nephrologists,many of whom are unfamiliar with the late onset form of the disease,identify patients who suffer from cystinosis." The CTNS gene is responsible for coding for the protein cystinosin, which isresponsible for transporting the amino acid cystine out of the lysosome incells. The accumulation of cystine is toxic to every cell, and can thereforeaffect all tissues and organs in the body, most notably the kidneys. "Nephropathic cystinosis is such a rare disease that it is likely that somecases have not been characterized in the adult population," said Amy Young,vice president and general manager of DaVita Clinical Research. "Ourbiorepository was designed and collected to enable this type of discoveryand we are pleased to embark on a project that has the potential to improveclinical outcomes for ESRD patients." About Nephropathic Cystinosis Nephropathic cystinosis comprises 95 percent of the diagnosed cases ofcystinosis, a rare, life-threatening metabolic lysosomal storage disorderthat causes toxic accumulation of cystine in all cells, tissues, and organsin the body. Elevated cystine leads to progressive, irreversible tissuedamage and multi-organ failure, including kidney failure, blindness, musclewasting and premature death. Nephropathic cystinosis is typically diagnosedin infancy and requires lifelong therapy. Left untreated, the disease isusually fatal by the end of the first decade of life. There are an estimated500 patients living in the United States with cystinosis, and 2,000worldwide. Cystine depletion is the primary treatment strategy for nephropathiccystinosis. However, poor adherence to therapy has been a major challengeresulting in poor sustained control of cystine levels, and patientsconsequently experience poor clinical outcomes, including kidneyinsufficiency leading to dialysis and kidney transplantation, muscle wastingand in some cases, premature death. Even brief interruptions in dailytherapy can permit toxic accumulation of cystine, exposing tissues torenewed, progressive deterioration. About Raptor Pharmaceuticals Raptor Pharmaceutical Corp. is a biopharmaceutical company focused ondeveloping and commercializing life-altering therapeutics that treat rare,debilitating and often fatal diseases. The company's first product,PROCYSBI(R) (cysteamine bitartrate) delayed-release capsules, is FDAapproved for the management of nephropathic cystinosis in adults andchildren 6 years and older. The European equivalent, PROCYSBI(R)gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate),received European Commission approval in September 2013 as an orphanmedicinal product for the treatment of proven nephropathic cystinosis formarketing in the European Union (EU). Raptor's pipeline also includes RP103in a Phase 2/3 trial for Huntington's disease and a Phase 2 trial innonalcoholic fatty liver disease in children. PROCYSBI was granted orphandesignation and exclusivity for nephropathic cystinosis in the U.S. and EUand RP103 has received U.S. orphan drug designation for Huntington'sdisease. For additional information, please visit www.raptorpharma.com. About DaVita Clinical Research DaVita Clinical Research (DCR), a wholly owned subsidiary of DaVitaHealthCare Partners Inc., uses its extensive, applied database andreal-world healthcare experience to assist pharmaceutical and medical devicecompanies in the design, recruitment, and completion of clinical trialsincluding retrospective and prospective pragmatic trials. DCR's scientificand clinical expertise spans the lifecycle of product development with morethan 150 client companies. DCR's Biorepository, Early Clinical Research unit(Phase I-IIa) and Clinical Development (Phase IIb through post-marketing)network of physicians and investigative sites, data research, HealthEconomics & Outcomes Research, and Medical Communications are focused onproviding world-class research in both complex/specialty populations andtherapeutic areas, and especially in CKD and ESRD populations. To learn moreabout DCR, visit www.davitaclinicalresearch.com. FORWARD-LOOKING STATEMENTS This document contains forward-looking statements as that term is defined inthe Private Securities Litigation Reform Act of 1995. These statements areindicated by words or phrases such as "believes," "expects," "anticipates,""estimates," "plans," "continuing," "ongoing", "projected" and similar wordsor phrases and relate to future events or our future results of operationsor future financial performance, including, but not limited to, statementsregarding the conduct and results of the collaboration with DaVita ClinicalResearch and the identification of patients with late onset cystinosis.These statements are only predictions and involve known and unknown risks,uncertainties and other factors, which may cause the Company's actualresults to be materially different from these forward-looking statements.Factors which may significantly change or prevent the Company'sforward-looking statements from fruition include: that Raptor may beunsuccessful in developing any products or acquiring products; that Raptor'stechnology may not be validated as it progresses further and its methods maynot be accepted by the scientific community; that Raptor is unable to retainor attract key employees whose knowledge is essential to the development ofits products; that unforeseen scientific difficulties develop with theCompany's process; that Raptor's patents are not sufficient to protectessential aspects of its technology; that competitors may invent bettertechnology; that Raptor's products may not work as well as hoped or worse,that the Company's products may harm recipients; and that Raptor may not beable to raise sufficient funds for development or working capital. As well,Raptor's products may never develop into useful products and even if theydo, they may not be approved for sale to the public. Raptor cautions readersnot to place undue reliance on any such forward-looking statements, whichspeak only as of the date they were made. Certain of these risks,uncertainties and other factors are described in greater detail in theCompany's filings from time to time with the Securities and ExchangeCommission (the "SEC"), which Raptor strongly urges you to read andconsider, including: Raptor's transition report for the four months endedDecember 31, 2012 on Form 10-KT filed with the SEC on March 14, 2013, asamended, and Raptor's Quarterly Report on Form 10-Q filed with the SEC onAugust 9, 2013, as amended, which is available free of charge on the SEC'sweb site at http://www.sec.gov. Subsequent written and oral forward-lookingstatements attributable to Raptor or to persons acting on its behalf areexpressly qualified in their entirety by the cautionary statements set forthin Raptor's reports filed with the SEC. Raptor expressly disclaims anyintent or obligation to update any forward-looking statements.CONTACT: Raptor Company Contact:

27% profits every 20 days?

This is what Nic Chahine averages with his options buys. Not selling covered calls or spreads... BUYING options. Most traders don't even have a winning percentage of 27% buying options. He has an 83% win rate. Here's how he does it.


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