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CheckOrphan NewsFlash January 15,…
1/15/2010
CheckOrphan NewsFlash for Friday, January 15, 2010: breaking news about rare diseases, orphan diseases, orphan drugs,…
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CheckOrphan NewsFlash January 11,…
1/11/2010
CheckOrphan NewsFlash for Monday, January 11, 2010: breaking news about rare diseases, orphan diseases, orphan drugs,…
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CheckOrphan NewsFlash December 16…
12/16/2009
CheckOrphan NewsFlash for Wednesday, December 16, 2009: breaking news about rare diseases, orphan diseases, orphan…
NewsFlash
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WORLD Symposium 2010 (Lysosomal…
10–12 February, 2010
Specific Aims of this meeting are to: 1.) Emphasize the strategies for,…
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2010 Neuromuscular Disorders…
26–27 February, 2010
Featuring a stimulating and progressive programme disseminating the most…
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Rare Disease Day Basel-Zurich
Sunday, 28 February, 2010
February 28, 2010, is RARE DISEASE DAY, a series of events in Basel and…
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Strategic Planning for Pulmonary…
8–9 March, 2010
Anticipated goals and use of results: Formulation of strategy for…
Events
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Gentium Announces an Abstract for Defibrotide in the Prevention of Hepatic VOD Has Been Granted the Prestigious Van Bekkum Award by the EBMT
Monday, February 8, 2010
VILLA GUARDIA, Italy -- Gentium S.p.A. (Nasdaq: GENT) today announced that the abstract titled, "Defibrotide prevents hepatic VOD and reduces significantly VOD-associated complications in children at high risk: final results of a prospective phase II/III multicentre study," has been awarded the Van
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Lux Biosciences Files for LUVENIQ™ Approval in US and Europe for Noninfectious Uveitis
Monday, February 8, 2010
JERSEY CITY, N.J. -- Lux Biosciences, Inc. today announced its submission of simultaneous regulatory filings to both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) seeking marketing approval for its investigational drug LUVENIQ™ (LX211; oral voclosporin) for
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AVI BioPharma's Drug Candidate AVI-5038 Receives European Orphan Drug Designation for Duchenne Muscular Dystrophy
Monday, February 8, 2010
BOTHELL, WA -- AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced that it received an orphan drug designation from the Committee for Orphan Medical Products of the European Medicines Agency (EMEA) for AVI-5038, a drug candidate being developed by AVI for the DMD is
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Talecris Biotherapeutics Receives Orphan Drug Designation for Aerosolized Alpha1-Proteinase Inhibitor to Treat Alpha1-Antitrypsin Deficiency
Monday, February 8, 2010
RESEARCH TRIANGLE PARK, N.C. -- Talecris Biotherapeutics, Inc. (Nasdaq: TLCR) announced today that it was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the development of an aerosol formulation of Alpha1-Proteinase Inhibitor (Human, A1PI) to treat congenital AA
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Vertex Announces Results From Phase 2a Trial of VX-809 Targeting the Defective Protein Responsible for Cystic Fibrosis
Monday, February 8, 2010
CAMBRIDGE, Mass. -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced results from a preliminary analysis of data from a 28-day Phase 2a clinical trial of VX-809 in patients with cystic fibrosis (CF) who are homozygous for the F508del mutationVX-809, an oral investigational Cystic
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Distinct Demographic Profiles Between Crohn's Disease and Ulcerative Colitis
Monday, February 8, 2010
Although inflammatory bowel disease (IBD) [comprising mainly Crohn's disease (CD) and ulcerative colitis (UC)] is thought to affect about 150 000 people in the United Kingdom, the prevalence of severe IBD is not known. Mortality following hospitalization for IBD is significant but little has been
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CREON(R) (Pancrelipase) Significantly Improves Fat Absorption in Children With Cystic Fibrosis
Monday, February 8, 2010
MARIETTA, Ga. -- Solvay Pharmaceuticals, Inc. announced today that Phase IIIb data published in the January issue of Clinical Therapeutics confirm that CREON® (pancrelipase) Delayed-Release Capsules significantly improves a key measure of fat absorption in children aged 7-11 years who have In
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Lung Rx LLC and mondoBIOTECH Holding AG Announce Today a Global Strategic Partnership Agreement for the Development of Aviptadil Platform
Friday, February 5, 2010
Silver Spring/Stans -- After completion by Mondobiotech of a Phase II study where inhaled Aviptadil was shown to have a unique safety profile and promising signals of long term efficacy over currently approved therapies in patients affected by Pulmonary Arterial Hypertension (PAH), United The plat
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New Malaria Vaccine Shows Promise in Children
Friday, February 5, 2010
CHICAGO -- A new vaccine showed promise at protecting young children from malaria, offering a potential new weapon against a disease that kills at least 1 million people each year, U.S. researchers said on WednesdayIn a study of 100 West African children aged 1 to 6, the experimental vaccine produ
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Protalix Announces Presentation of Phase III Taliglucerase Alfa Data at WORLD Lysosomal Disease Network
Friday, February 5, 2010
CARMIEL, Israel -- Protalix Biotherapeutics, Inc. (NYSE- Amex: PLX) today announced that data from its pivotal Phase III trial of taliglucerase alfa in patients with Gaucher disease will be presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010, February 10-12, 2010
