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Latest Orphan drug Stories

2014-05-21 00:20:51

EPI-743 in phase 2B/3 development for Leigh syndrome MOUNTAIN VIEW, Calif., May 21, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the Japanese Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Designation to EPI-743 for the treatment of Leigh syndrome. EPI-743 is currently in phase 2B development for the treatment of Leigh syndrome. A phase 2B randomized double-blind placebo-controlled trial in children with Leigh syndrome is fully enrolled in the...

2014-05-15 12:29:33

LONDON, May 15, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report:Global Markets for Orphan Drugshttp://www.reportbuyer.com/pharma_healthcare/drug_discovery/global_markets_orphan_drugs.htmlINTRODUCTIONSTUDY OBJECTIVESThe orphan drug market is one of the fastest-growing segments in the pharmaceutical and biotechnology industry. BCC's goal in conducting this study is to provide an overview of the current and future global market for orphan drugs. The key...

2014-05-14 23:09:18

New Kauffman Foundation paper highlights solutions to speed patients’ access to drugs with therapeutic value for more than one disease. Kansas City, MO (PRWEB) May 14, 2014 For patients with rare medical conditions, the typical 12 to 15 years it takes to bring a new drug to market may be too long to wait. Repurposed drugs – those already approved for a specific condition that can be used to treat another – offer a solution: Because their safety profile is already established, they...

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...


Word of the Day
snash
  • To talk saucily.
  • Insolent, opprobrious language; impertinent abuse.
This word is Scots in origin and probably imitative.