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Total Orphan Drugs
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Exclusive Interview with Dr Jonathan Morton, Oxford PharmaGenesis™ Ltd Speaking at The World Orphan Drug Congress http://ow.ly/2NcrW3
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Asfotase Alfa Granted Orphan Drug Designation in Japan - September 8, 2014 Alexion Pharmaceuticals, Inc. today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation (ODD) to asfotase alfa for the treatment of patients with hypophosphatasia (HPP), a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization. Patients with HPP can experience a range of [] http://ow.ly/2N9l81
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Genzyme Prices Gaucher Disease Drug at $310,250 a Year - Genzyme's recently approved orphan drug, Cerdelga, will be priced at $310,250 a year The Masachusetts-based biotech company Genzyme, owned by pharmaceutical giant Sanofi, will be pricing its rare disease drug for the treatment of Gaucher's disease at $310,250 a year. The US Food and Drug Administration approved the drug and granted it orphan drug status [] http://ow.ly/2MZnr1
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The Top 10 Most Researched Rare Diseases - With the ALS Ice Bucket challenge currently generating a large amount of interest, ALS itself has recently become a very well-known rare disease. However, the key question within the Orphan Drug industry is 'which other rare diseases now have the highest number of companies investing money into research for them?' This eBook explores the Top 10 [] http://ow.ly/2MZbkc
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Family Photo Scans: The Future of Rare Disease Diagnosis - Scientists at Oxford University have developed a software that can detect the risk that a child has a genetic disorder by scanning family photos Scientists at the University of Oxford have developed a revolutionary new software that can help diagnose someone's risk of having a rare genetic disorder by scanning family photographs. Most rare diseases [] http://ow.ly/2MVV1d
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ALS Ice Bucket Challenge: Where is the Money Going? - The ALS Association has received $106 million in ice bucket challenge donations, but what exactly will the money be used for? The ALS Association has enjoyed astronomical levels of success with their viral ice bucket challenge campaign to raise awareness and funds for ALS. From the 29th of July to the 2nd of September, the [] http://ow.ly/2MRWRM
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Arena Pharmaceutical's Pulmonary Arterial Hyeprtension Drug Receives Orphan Drug Status http://ow.ly/2MQVHN
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