Latest Orphan drug Stories

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)...

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...

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,...

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...

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...

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...

QED Clinical Services, a global niche CRO operating in more than 40 countries, has announced that it will address the increasing demand of biotech and pharma companies to develop drugs in rare disease areas by launching OrphanReach, a division dedicated to this increasingly important research area. Milton Keynes, UK (PRWEB) August 26, 2012 The company's CEO, Thomas Ogorka, summarised QED's strategy moving forward: "We can clearly see a strong industry trend for orphan drug...

PHILADELPHIA, Aug. 22, 2012 /PRNewswire/ -- The Intellectual Property & Science business of Thomson Reuters, the world's leading provider of intelligent information for businesses and professionals, today released the results of its study on orphan drugs, developed to treat rare diseases, finding that they have the potential to generate as much lifetime revenue as drugs used for more common health conditions. The findings are featured in the Thomson Reuters paper, "The Economic...

NEW YORK and JERUSALEM, July 23, 2012 /PRNewswire/ -- Chiasma (pronounced key-azma), Inc., a privately held biopharma company, announced that it closed a $38.5 million equity financing co-led by new investor Abingworth and current investor MPM Capital that also included investments from existing shareholders 7 Med Health Ventures, ARCH Venture Partners, F3 Ventures and Fredric Price, Chiasma's Chairman & CEO. The new investment (the "D" round) is expected to provide funding for...

RESEARCH TRIANGLE PARK, N.C., July 17, 2012 /PRNewswire/ -- Grifols, a global healthcare company based in Barcelona, Spain, today announced plans to initiate a safety trial of a novel, inhaled formulation of alpha1-proteinase inhibitor [Human] later this year. In April, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for Grifols' inhaled alpha1 formulation as a treatment for cystic fibrosis, an inherited disease that can cause life-threatening lung...