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Latest Orphan drug Stories

2014-05-12 08:32:50

CLEVELAND, May 12, 2014 /PRNewswire-USNewswire/ -- Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted Orphan Drug Designations for its lead investigational therapies by the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development. Following the successful close of seed financing in late 2013, Abeona is raising funds...

2014-05-07 08:29:17

Winners include Teva's Coxapone, a specialty drug for the treatment of chronic disease, and Corcepts' KORLYM, an orphan drug for a rare condition NEW YORK, May 7, 2014 /PRNewswire/ -- PHARMACEUTICAL EXECUTIVE magazine's June cover story and the magazine's "iDigital Marketing Winning Brands," an interactive eBook available for free in the iTunes Store https://itunes.apple.com/us/app/pharma-sci/id568221918?mt=8 features the publication's eighth annual Brand of The Year awards. This...

2014-04-30 08:31:42

PURIXAN(TM) (mercaptopurine) Oral Suspension Finally Makes Accurate Dosing Simple for Healthcare Professionals to Achieve--Especially with Children FRANKLIN, Tenn., April 30, 2014 /PRNewswire/ -- Rare Disease Therapeutics, Inc. (RDT), the producer of orphan pharmaceuticals for the treatment of rare diseases, announced today that they have received FDA approval for PURIXAN(TM) (mercaptopurine) 20 mg/mL oral suspension---a new, easier-to-dose, liquid form of an established treatment for...

2014-04-29 20:23:44

LONDON, April 29, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report:The World Market for Orphan Drugshttp://www.reportbuyer.com/pharma_healthcare/therapeutic/world_market_orphan_drugs_1.htmlIn recent years, there has been a substantial increase in the number of drugs available to treat rare ("orphan") diseases. This comprehensive report, The World Market for Orphan Drugs, looks at this trend and provides estimates for the market opportunity in the U.S.,...

2014-04-24 08:36:03

The company releases white paper focused on a growing trend in the industry RESEARCH TRIANGLE PARK, N.C., April 24, 2014 /PRNewswire/ -- Marken announced today the release of a new white paper which identifies supply chain solutions for the rapidly growing orphan drug market. http://photos.prnewswire.com/prnvar/20110930/NY78064LOGO The production of orphan drugs, which are used to treat rare diseases, is increasing and will continue to increase with a compound annual growth...

2014-04-17 00:21:10

DUBLIN, April 17, 2014 /PRNewswire/ -- Heart Metabolics Limited, a new Irish biotechnology company focused on development of drugs for cardio-metabolic diseases, has announced the completion of a $20 million Series A financing from venBio, Seroba Kernel Life Sciences, Brandon Capital Partners (on behalf of AustralianSuper) and AshHill. The proceeds from this financing have supported Heart Metabolics Limited in acquiring a Phase 3-ready drug candidate and related preclinical drug...

2014-04-10 23:00:56

Symposium to Highlight How Legislation in Europe and U.S. Can Help Bring Medicines to Market Washington (PRWEB) April 10, 2014 “Regulatory Challenges for Orphan Medicines,” a symposium that features presentations by a number of industry experts, will take place during the DIA 2014 50th Annual Meeting at the San Diego Convention Center from June 15 to 19. The symposium, held June 18 at 3:30 p.m., will focus on the regulatory environment and trends for orphan medicinal products around...

2014-04-07 04:21:16

MADISON, Wis., April 7, 2014 /PRNewswire/ -- The European Medicines Agency (EMA) has granted Minor Use Minor Species (MUMS) designation for Nexcyon's adrenocorticotropin (ACTH), a synthetic peptide for the diagnosis of hypoadrenocorticism in dogs. Hypoadrenocorticism, also known as Addison's Disease, can be difficult to diagnose without the aid of an ACTH injection. Nexcyon has developed a proprietary form of synthetic ACTH specifically formulated for use in dogs. Presently, when...

2014-04-02 23:32:53

Cambridge Biomedical Inc. announces that their Chief Operating Officer, Dr. John Reddington, will be attending the 4th annual World Orphan Drug Congress in Washington D.C. April 23-25th, 2014 Boston, MA (PRWEB) April 02, 2014 Cambridge Biomedical’s experience with bioanalytical assay development, optimization, validation and diagnostic testing has proved instrumental in assisting biotechnology and pharmaceutical companies that are focusing on therapies for orphan and ultra-rare disease...

2014-03-31 20:22:33

DUBLIN, April 1, 2014 /PRNewswire/ -- Research and Markets ( http://www.researchandmarkets.com/research/5q5tcr/global_orphan) has announced the addition of the "Concise Analysis of the International Orphan Drug Market Outlook 2018" [http://www.researchandmarkets.com/research/5q5tcr/global_orphan ] report to their offering. (Logo: http://photos.prnewswire.com/prnh/20130307/600769 ) The global pharmaceutical industry has been experiencing a slow growth in recent...


Word of the Day
Cthulhu
  • A gigantic fictional humanoid alien god being described with a head resembling an octopus and dragon wings and claws, around whom an insane cult developed.
  • Pertaining to the mythos of Cthulhu and additional otherworldly beings created by H. P. Lovecraft or inspired by his writings and imitators.
This word was invented in 1926 by H.P. Lovecraft for his short story, 'The Call of Cthulhu.' 'Cthulhu' may be based on the word 'chthonic,' which in Greek mythology refers to the underworld.
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