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Latest Orphan drug Stories

2014-07-07 08:27:35

ROCKVILLE, Md., July 7, 2014 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a clinical-stage pharmaceutical company developing therapeutics for the treatment of cancer and other diseases, announced today that its orally-active, Aurora A/angiogenic kinase inhibitor, ENMD-2076, has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma (HCC). http://photos.prnewswire.com/prnvar/20140706/124566...

2014-07-02 12:26:27

LONDON, July 2, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report: Global Orphan Drugs Market 2014-2018http://www.reportbuyer.com/pharma_healthcare/therapeutic/global_orphan_drugs_market_2014_2018.html TechNavio's analysts forecast the Global Orphan Drugs market to grow at a CAGR of 5.67 percent over the period 2013-2018. One of the key factors contributing to this market growth is the shorter development timelines. The Global Orphan Drugs market has also...

2014-06-24 12:27:50

ST. PETERSBURG, Fla., June 24, 2014 /PRNewswire-USNewswire/ -- InformedDNA announced today a strategic new collaboration with Edimer Pharmaceuticals to address a critical research need for patients with X-linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder. The partnership will allow InformedDNA to identify patients with XLHED and support Edimer's clinical programs. Under the agreement, InformedDNA has developed a genetic counseling program for patients and...

2014-06-23 04:20:30

PARIS, June 23, 2014 /PRNewswire/ -- Pharnext SAS today announced that the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted orphan drug designation to PXT-3003 for the treatment of Charcot-Marie-Tooth disease type 1A (CMT 1A). Highlights

2014-06-20 23:08:51

In an environment where orphan drug development is on an all-time high and Contract Research Organizations are in demand, orphan reach, who pioneer a unique global patient access solution, has expanded its presence to the US. Audubon, New Jersey (PRWEB) June 20, 2014 orphan reach, the “rare” Contract Research Organization (CRO) focusing on global orphan drug development, has today announced the opening of new offices in New Jersey, USA to target the growing opportunities in the orphan...

2014-06-18 12:36:30

LONDON, June 18, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report: Orphan Drugs for CNS Diseases: R&D and Market 2014-2024http://www.reportbuyer.com/pharma_healthcare/therapeutic/orphan_drugs_cns_diseases_rd_market_2014_2024.html Report DetailsUncommon nerve disorders - new study explains technological and commercial prospectsWhat's the future of medicines for rare central nervous system (CNS) disorders? Visiongain's new report gives you predictions...

2014-06-14 23:01:01

Hot topics on Wednesday, June 18 SAN DIEGO (PRWEB) June 14, 2014 Patient perspective in clinical trials, efforts of the Food and Drug Administration (FDA) to increase the number of regulators overseas, the emerging practice of risk-based monitoring in clinical trials, and challenges in orphan drug regulation will be discussed Wednesday, June 18, at the DIA 2014 50th Annual Meeting at the San Diego Convention Center. Patient Perspective to Drive Enrollment in Clinical Trials “The...

2014-06-10 08:31:42

DOYLESTOWN, Pa., June 10, 2014 /PRNewswire/ -- On June 2, 2014 Makindus, Inc. a specialty pharmaceutical company, received orphan drug designation for their lead product MI-100 from the U.S. Food and Drug Administration Office of Orphan Products Development for the treatment of Stargardt's Disease. "The FDA's approval of our orphan drug designation application for MI-100 to treat Stargardt's disease is a key milestone for the company and we look forward to initiating our Phase 3...

2014-06-09 00:21:32

EPI-743 in phase 2B development for Leigh syndrome MOUNTAIN VIEW, Calif., June 9, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome. Vatiquinone is the International Nonproprietary Name (INN) for Edison's EPI-743. The INN is a unique international name issued by the World Health Organization, which is used to identify the active pharmacological ingredient in a drug. This is...

2014-06-04 23:05:47

Sessions to provide international perspectives and reveal new collaborative approaches. Washington (PRWEB) June 04, 2014 The world’s foremost drug industry regulators will address urgent topics surrounding regulatory guidance, such as the challenges and future of the Food and Drug Administration (FDA) and its international counterparts, at the DIA 2014 50th Annual Meeting to be held at the San Diego Convention Center from June 15 to 19. “We are honored and excited to have the...


Word of the Day
mundungus
  • A stinking tobacco.
  • Offal; waste animal product; organic matter unfit for consumption.
This word comes from the Spanish 'mondongo,' tripe, entrails.