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CheckOrphan NewsFlash April 30,…
4/30/2010
CheckOrphan NewsFlash for Tuesday, March 30, 2010: breaking news about rare diseases, orphan diseases, orphan drugs,…
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CheckOrphan NewsFlash April 9,…
4/09/2010
CheckOrphan NewsFlash for Friday, April 9, 2010: breaking news about rare diseases, orphan diseases, orphan drugs, and…
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CheckOrphan NewsFlash April 1,…
4/01/2010
CheckOrphan NewsFlash for Thursday, April 1, 2010: breaking news about rare diseases, orphan diseases, orphan drugs,…
NewsFlash
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Men 2010: 12th International…
16–18 September, 2010
This two-day meeting will provide a forum for educating basic and clinical…
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Ribonuclease H2010
20–22 September, 2010
We expect to bring together scientists interested in making as much…
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Third Annual Ultra Orphan…
27–29 September, 2010
Centric Health Resources Presents Third Annual Ultra Orphan Conference…
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Brain, Blood and Iron: Joint…
1–2 October, 2010
The overall objectives of this workshop are to (1) define NA and NBIA…
Events
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Ipilimumab Receives FDA Priority Review Designation for Adult Patients With Previously Treated Advanced Melanoma
Thursday, August 19, 2010
PRINCETON, N.J. -- Bristol-Myers Squibb Company (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted, for filing and review, the Biologics License Application (BLA) for ipilimumab for the treatment of adult patients with advanced melanoma who have been The comp
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NephRx Corp. Receives Orphan Drug Designation for NX001 for Prevention of Delayed Kidney Graft Function
Thursday, August 19, 2010
KALAMAZOO, Mich. -- NephRx Corporation today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product NX001 for the prevention of delayed graft function (DGF) in renal transplant recipientsNX001 is a kidney growth factor peptide that has de
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ImmunoGen, Inc.Announces Orphan Drug Designation Granted to Lorvotuzumab Mertansine for Treatment of Small-Cell Lung Cancer
Thursday, August 19, 2010
WALTHAM, Mass. -- ImmunoGen, Inc. (Nasdaq: IMGN), a biotechnology company that develops antibody-based targeted anticancer products, today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to its lorvotuzumab mertansine (formerly IMGN901) product candidate
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Lilly Halts Development of Semagacestat for Alzheimer's Disease Based on Preliminary Results of Phase III Clinical Trials
Thursday, August 19, 2010
INDIANAPOLIS -- Eli Lilly and Company (NYSE: LLY) will halt development of semagacestat, a gamma secretase inhibitor being studied as a potential treatment for Alzheimer's disease, because preliminary results from two ongoing long-term Phase III studies showed it did not slow disease progression T
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Global Media Campaign Finds 'Hidden' Children With Rare, Fatal Aging Disorder
Thursday, August 19, 2010
ATLANTA, GA -- Spectrum, a health and science communications firm, announced the results of a global awareness campaign that "found" 24 percent more children identified with a rare, fatal, and rapid aging disease called ProgeriaThe findings are being shared at a Centers for Disease Control nationa
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Idebenone Does Not Improve Friedreich's Ataxia in Pediatric Patients
Thursday, August 19, 2010
Idebenone does not appear to improve neurologic function in pediatric patients with Friedreich's ataxia, according to the findings of a randomized phase 3 trialDavid R. Lynch, MD, PhD, with the University of Pennsylvania School of Medicine in Philadelphia, and colleagues report their findings in t
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AMT's Marketing Authorisation Application for Glybera(R) Progressing on Schedule
Thursday, August 19, 2010
AMSTERDAM, The Netherlands -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that the Marketing Authorisation Application (MAA) for Glybera(R) remains on schedule following meetings with the European Medicine Agency (EMA) concerning T
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Biogen Idec and Knopp Neurosciences Announce License Agreement for Late-Stage ALS Drug Candidate 08/18/2010
Thursday, August 19, 2010
WESTON, MA, and PITTSBURGH, PA -- Biogen Idec (NASDAQ: BIIB) and Knopp Neurosciences today announced they have entered into an exclusive, worldwide license agreement under which Biogen Idec will develop and commercialize KNS-760704 (dexpramipexole) for the treatment of amyotrophic lateral sclerosis
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MDA Awards $14.1 Million in New Grants to Advance Treatments for Muscle and Nerve Diseases
Wednesday, August 18, 2010
TUCSON, Ariz. -- The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants"Thanks to generous public support of MDA, we're able to keep fu
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BioMarin Acquires ZyStor Therapeutics, Inc.
Wednesday, August 18, 2010
NOVATO, Calif. -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disordersZyStor's lead product candidat
