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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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Open-Label Continuation Study Supports Long-Term Efficacy of Xenazine (Tetrabenazine) for the Treatment of Chorea Associated With Huntington's Disease
Monday, March 8, 2010
DEERFIELD, Ill -- Lundbeck Inc. today announced the presentation of results from an open-label extension study of Xenazine® (tetrabenazine) for the treatment of chorea associated with Huntington's disease (HDData from this study demonstrated that after an 80-week treatment period, subjects tre
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University of Chicago and Ataxia Foundation Team Up for Annual Scientific and Patient Meetings
Monday, March 8, 2010
The National Ataxia Foundation (NAF), in conjunction with the University of Chicago program in pathobiology and translational neuroscience, will hold its third annual Ataxia Investigators Meeting March 9-11, 2010, at the Hyatt Regency O'Hare, in Rosemont, IllA three-day meeting for patients, careg
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ISIS Initiates Phase 1 Clinical Trial of ISIS-SOD1Rx in Patients With ALS
Monday, March 8, 2010
CARLSBAD, Calif. -- Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced today that it has initiated a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig's disease also known as familial amyotrophic lateral sclerosis (ALSApproximately 20 percent of all familia
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Peptimmune Announces Second Grant of a United States Patent for PI-2301 Peptide Copolymer for Multiple Sclerosis
Monday, March 8, 2010
CAMBRIDGE, Mass. -- Peptimmune, Inc. a privately held biotechnology company, announced today the grant of US Patent Number 7,655,221 (the '221 patent) which protects the target product profile for its PI-2301 peptide copolymer for the treatment of multiple sclerosis, and other autoimmune diseasesT
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NICE Recommends Infliximab and Adalimumab for the Treatment of Severe, Active Crohn's Disease
Friday, March 5, 2010
In final draft guidance published today (4 March) NICE has recommended infliximab and adalimumab as treatment options for people with severe, active Crohn’s disease whose condition has not responded to conventional therapy, or who are intolerant of or have contraindications to conventional C
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NICE Set to Recommend Rituximab for Relapsed or Refractory Chronic Lymphocytic Leukaemia
Friday, March 5, 2010
The National Institute for Health and Clinical Excellence (NICE) has today (4 March) issued final draft guidance recommending the drug rituximab(MabThera) as a treatment for certain patients with relapsed or refractory chronic lymphocytic leukaemiaThis draft is now with consultees who have the opp
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CSL Behring Receives FDA Approval of Hizentra(TM), First 20 Percent Subcutaneous Immunoglobulin Therapy
Friday, March 5, 2010
King of Prussia, PA -- CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Hizentra™, Immune Globulin Subcutaneous (Human), 20% Liquid, for treating patients diagnosed with primary immunodeficiency (PIA once weekly immunoglobulin (I
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Rare Disorder Blood Samples to Be Sent to UK
Friday, March 5, 2010
ISLAMABAD -- The Ministry of Health has decided to send blood samples of seven children suffering from a rather uncommon disorder to Saint John’s Dermatology Centre in London for tests, because the country does not have facilities for diagnosing certain skin diseasesThe children from Balochi
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Pakistan Medical Research Council Formulates Guidelines for Management of Snakebite
Friday, March 5, 2010
Islamabad -- The Pakistan Medical Research Council (PMRC) has launched the world’s first comprehensive snakebite protocol called 'A2 Snakebite Management in Asia and Africa' for use by doctors to treat snakebite in the key areas of Asia and AfricaSnakebite has been described as a 'neglected
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PTC Therapeutics and Genzyme Corporation Announce Preliminary Results From the Phase 2b Clinical Trial of Ataluren for Nonsense Mutation Duchenne/Becker Muscular Dystrophy
Friday, March 5, 2010
SOUTH PLAINFIELD, N.J. & CAMBRIDGE, Mass. -- PTC Therapeutics, Inc. and Genzyme Corporation (Nasdaq: GENZ) today announced preliminary results from the Phase 2b clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation Duchenne/Becker Muscular Dystrophy (nmDBMD
