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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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AA&MDSIF Bone Marrow Failure…
11–12 March, 2010
Registration is now open for the second Bone Marrow Failure Disease…
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Brain, Blood, and Iron: Advances…
13–14 March, 2010
The overall objectives of this workshop are to: (1) define NA and NBIA…
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Diamond Blackfan Anemia…
13–15 March, 2010
This conference is expected to generate immediate and long-term…
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Interdisciplinary Training…
15–16 March, 2010
The goal of this meeting is to mentor the career development of a “…
Events
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Happy Birthday Seth
Wednesday, March 10, 2010
SPEARFISH -- From a distance Seth looks like any tired 8-year-old boy taking a nap on a couchA “Thomas the Train” blanket covers him as his head rests on a pillowThen you notice it. A box of medical supplies is lying on a table in front of the brown-haired child. A tube runs to his no
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The Watery World of a Little Figtree Boy
Wednesday, March 10, 2010
A pesky little molecular glitch makes life tough for this little boy whose thirst is so fierce he drinks in his sleepLiam Walter, 5, of Figtree, has a disorder known as nephrogenic diabetes insipidus, which interferes with his kidney's ability to conserve waterSufferers urinate on average every h
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Actelion Receives FDA Complete Response Letter for Zavesca (Miglustat) for the Treatment of Niemann-Pick Type C Disease
Wednesday, March 10, 2010
ALLSCHWIL/BASEL, SWITZERLAND -- Actelion Ltd (SIX: ATLN) announced today that the company has received a complete response letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for Zavesca® (miglustat) for the treatment of progressive The FDA
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Ark Withdraws Cerepro(R) MAA After SAG-O Requests Further Trial Before Approval
Wednesday, March 10, 2010
London, UK -- The Board of Ark Therapeutics Group plc ("Ark" or "the Company") today announces an update relating to the Marketing Authorisation Application ("MAA") for its brain cancer treatment, Cerepro®Following a presentation to the EMEA's Scientific Advisory Group on Oncology ("SAG-O") as
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Abbott Enhances Pharmaceutical Pipeline With Acquisition of Facet Biotech
Wednesday, March 10, 2010
ABBOTT PARK, Ill., and REDWOOD CITY, Calif. -- Abbott (NYSE: ABT) and Facet Biotech Corporation (Nasdaq: FACT) announced today a definitive agreement for Abbott to acquire Facet, enhancing Abbott's early- and mid-stage pharmaceutical pipelineAbbott will acquire Facet for $27 per share in cash for
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At an FDA Workshop, a Mom Looks for Help
Wednesday, March 10, 2010
CLAREMONT, Calif. -- Among the 14 groups at the Food and Drug Administration workshop that filed applications for orphan drug designation, Chris Hempel—part of a small team—stood out in her long-sleeved T-shirt and jeansThe corporate potential applicants at the event tended to guard th
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Omeros Licenses PDE7 Compounds for Parkinson's Disease Program
Wednesday, March 10, 2010
SEATTLE -- Omeros Corporation (Nasdaq: OMER) today announced that it has obtained an exclusive license to compounds from Asubio Pharma Co., Ltd. for use in its PDE7 program, which is focused on the treatment of movement disordersOmeros has demonstrated a previously unknown link between phosphodies
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FDA Advisory Committee Recommends Approval of InterMune's Esbriet(R) (Pirfenidone) for Idiopathic Pulmonary Fibrosis
Wednesday, March 10, 2010
BRISBANE, Calif. -- InterMune, Inc. (Nasdaq: ITMN) announced today that the U.S. Food and Drug Administration's (FDA) Pulmonary-Allergy Drugs Advisory Committee (PADAC) voted 9-3 to recommend approval of Esbriet® (pirfenidone) for the treatment of patients with idiopathic pulmonary fibrosis (I
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New Evidence Further Validates Ketone Body Therapy as an Effective Approach in Managing Alzheimer's Disease
Wednesday, March 10, 2010
THESSALONIKI, Macedonia, Greece -- Accera, Inc., a biotechnology company delivering breakthrough therapies in central nervous system diseases, announced data which showed that augmentation with ketone bodies significantly improved cognitive function in Alzheimer's disease (AD) patientsAn early fea
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Cord Blood America's New Medical Director Says Parkinson's, Diabetes Among Diseases to Be Treated With Umbilical Cord Blood Stem Cells
Wednesday, March 10, 2010
LAS VEGAS -- Cord Blood America, Inc. (OTC Bulletin Board: CBAI), the umbilical cord blood stem cell preservation company (http://www.cordblood-america.com), is focused on bringing the life saving potential of stem cells, a biological insurance policy, to families nationwide and internationallyIts
