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Last updated on April 18, 2014 at 15:17 EDT

Latest Orphan drug Stories

2013-01-03 08:25:15

SUNNYVALE, Calif., Jan. 3, 2013 /PRNewswire/ -- Icon Bioscience, Inc., (IBI), a privately held biopharmaceutical company that specializes in the development and commercialization of novel ophthalmic pharmaceuticals, today announced that melphalan intraocular injection, the company's investigational product for the treatment of retinoblastoma, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA). Orphan Drug Designation is generally granted to drugs or...

2012-12-25 12:20:05

WASHINGTON, Dec. 25, 2012 /PRNewswire-USNewswire/ -- Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations. He did this in partnership with patients and patient advocates who...

2012-11-14 08:31:29

AMES, Iowa, Nov. 14, 2012 /PRNewswire/ -- NewLink Genetics Corporation (Nasdaq: NLNK) today announced that the European Commission (EC) has designated HyperAcute-Pancreas® Immunotherapy (algenpantucel-L) as an orphan medicinal product under Regulation (EC) No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products. NewLink filed the application for orphan designation in June 2012. A positive opinion was adopted by the Committee for Orphan...

2012-11-12 08:28:33

EXTON, Pa., Nov. 12, 2012 /PRNewswire/ -- Morphotek(® )Inc., a subsidiary of Eisai Inc., announced today that the United States Food and Drug Administration (FDA) has granted orphan drug designation to its investigational cancer drug, amatuximab (MORAb-009) for the treatment of malignant pleural mesothelioma, the most common form of mesothelioma, which accounts for around 2,500 cases a year in the United States. This disease affects the pleura, which is the thin balloon shaped lining of...

2012-11-01 07:31:02

SUNNYVALE, Calif., Nov. 1, 2012 /PRNewswire/ -- Amarantus BioSciences, Inc. (OTCQB: AMBS), a biotechnology company developing new treatments and diagnostics for Parkinson's disease and Traumatic Brain Injury centred on its proprietary anti-apoptosis therapeutic protein MANF, today announced that it intends to pursue the identification of one or multiple therapeutic indications for its lead therapeutic candidate MANF that could lead to the therapy receiving orphan drug designation(s) with the...

2012-10-18 19:21:00

HORSHAM, Pa., Oct. 18, 2012 /PRNewswire/ -- A panel of parents who became patient advocates after their children were diagnosed with rare diseases will discuss their struggles and successes in navigating the drug development process on Monday at a Washington, D.C., conference sponsored by DIA and the National Organization for Rare Disorders (NORD). The work carried out by these parents through rare disease associations can be one of the most important factors in getting lifesaving help...

2012-10-17 11:24:20

HORSHAM, Pa., Oct. 17, 2012 /PRNewswire/ -- The 2012 U.S. Conference on Rare Diseases and Orphan Products will bring together the rare disease and orphan product communities to collaborate on and advance product research, drug development and access to treatments to help the 30 million Americans suffering from rare diseases. Sponsored by DIA and the National Organization for Rare Disorders (NORD), the conference will be held Oct. 22-24 at the Capital Hilton hotel in Washington, D.C. Among...

2012-10-09 04:01:52

DIA and the National Organization for Rare Disorders (NORD), in collaboration with FDA, NIH, EURORDIS, and the Duke Department of Pediatrics, will host the 2nd Annual US Conference on Rare Diseases & Orphan Products: Shaping the Future Now from October 22-24 in Washington, DC. Horsham, PA (PRWEB) October 08, 2012 DIA and the National Organization for Rare Disorders (NORD), in collaboration with FDA, NIH, EURORDIS, and the Duke Department of Pediatrics, will host the [2nd Annual US...

2012-10-08 11:24:04

RESEARCH TRIANGLE PARK, N.C., Oct. 8, 2012 /PRNewswire/ -- Grifols, a global healthcare company based in Barcelona, Spain, announced today that the first patient has been enrolled and dosed in a phase II study evaluating the safety and tolerability of Alpha-1 HC aerosol in patients with cystic fibrosis. Cystic fibrosis is an inherited disease that causes progressive decline in lung function, including acute episodes of worsening respiratory symptoms and potentially fatal lung infections....

2012-09-25 23:02:25

Get 50% off on reports on Chinese Markets for Cardiovascular Drugs, Treatment Drugs for Arthritis, Asthma, Leukemia, Tuberculosis, Kidney & Skin Cancer, Chinese Markets for Digestive Remedies, Anti-infectious Disease Drugs, Pain and Fever Relief Drugs and more. Also, explore the latest developments of Drug Discovery in Rare Diseases. Dallas, TX (PRWEB) September 25, 2012 China's demand for drugs has grown at a fast pace in the past decade. In the next five years, both production and...