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CheckOrphan NewsFlash, March 2,…
3/02/2010
CheckOrphan NewsFlash for Tuesday, March 2, 2010: breaking news about rare diseases, orphan diseases, orphan drugs, and…
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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,…
NewsFlash
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Interdisciplinary Training…
15–16 March, 2010
The goal of this meeting is to mentor the career development of a “…
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ICORD 2010: Sixth International…
18–20 March, 2010
The VI International Conference on Rare Diseases and Orphan Drugs (ICORD…
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Improving Musculoskeletal Outcomes…
7–9 April, 2010
The goals of this workshop are to (1) assess the state of knowledge…
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Third NIH Workshop on Gaucher…
8–9 April, 2010
Workshop goals are to (1) determine the appropriate human, animal, or cell…
Events
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Molecular Basis for Pseudomonas Aeruginosa Persistent Infections in CF Patients
Friday, March 12, 2010
New research reveals Small Colony Variants (SCVs) of P. aeruginosa to be a hallmark of chronic infection in cystic fibrosis (CF) patients. Results, published March 12th in the open-access journal PLoS Pathogens, suggest that SCV-mediated persistence might be a good target for antimicrobial Cystic
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FDA Grants Orphan Drug Status to Ciprofloxacin Dry Powder Inhaler for the Treatment of Cystic Fibrosis
Thursday, March 11, 2010
Berlin -- Bayer Schering Pharma AG, Germany, announced today that an orphan drug designation has been granted by the U.S. Food and Drug Administration (FDA) for the ciprofloxacin dry powder inhaler (DPI) for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in cystic A s
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FDA Approves First New Inhaled Antibiotic for CF in More Than a Decade
Thursday, February 25, 2010
BETHESDA, Md. -- The U.S. Food and Drug Administration approved an important new inhaled antibiotic called Cayston® (aztreonam for inhalation solution) today for the treatment of cystic fibrosisThe drug was made possible by significant support from the Cystic Fibrosis Foundation, including a $
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North Carolina Couple Finds Hope for a Healthy Baby Through Technology That Screens Embryos for Genetic Disorders
Thursday, February 25, 2010
RALEIGH, N.C. -- After a devastating loss of their five month old son to a genetically linked disease, a North Carolina couple finds hope for a healthy baby through an innovative technology called Preimplantation Genetic Diagnosis (PGDUsing embryo biopsy, PGD allows geneticists to screen for disor
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U.S. Food and Drug Administration Approves Cayston(R) (Aztreonam for Inhalation Solution) for the Improvement of Respiratory Symptoms in Cystic Fibrosis Patients With Pseudomonas Aeruginosa
Wednesday, February 24, 2010
FOSTER CITY, Calif. -- Gilead Sciences, Inc. (Nasdaq:GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Cayston(R)(aztreonam for inhalation solution) as a treatment to improve respiratory symptoms in cystic fibrosis (CF) patients with Cayston'
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Defective Signaling Pathway Sheds Light on Cystic Fibrosis
Monday, February 15, 2010
In a study that could lead to new therapeutic targets for patients with the cystic fibrosis, a research team from the University of California, San Diego School of Medicine has identified a defective signaling pathway that contributes to disease severityIn the study, published in the journal Natur
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Kamada Announces Enrollment of First Patient in Its Pivotal Study for Inhaled AAT in Europe
Tuesday, February 9, 2010
NESS ZIONA, Israel -- Kamada, a bio-pharmaceutical company engaged in the development, manufacturing and marketing of specialty life-saving therapeutics, announced today that it has enrolled the first patient into its pivotal clinical trial with its new breakthrough compound of inhaled alpha-1 The
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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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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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NIGMS Awards Contract to Expand Human Genetic Cell Repository
Tuesday, January 26, 2010
The National Institute of General Medical Sciences (NIGMS) has awarded a $27 million, five-year contract to the Coriell Institute for Medical Research in Camden, N.J., to continue and expand operation of the NIGMS Human Genetic Cell Repository (HGCR). Under the new contract, the HGCR plans to "The
