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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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8th Annual Encephalitis Conference
Monday, 16 August, 2010
Encephalitis Global, Inc. is happy to invite FACES (Friends And Caregivers…
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David W. Smith Workshop on…
27 August – 1 September, 2010
The goal of the Smith Workshop is to bring together a diverse group of…
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The 15th International Symposium…
29 August – 1 September, 2010
Studies on the sinusoidal cells are becoming increasingly important in…
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Immunotherapy in Pediatric…
9–10 September, 2010
Goals of this conference are to (1) enhance collaboration and cross-…
Events
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Living With a Rare Genetic Disease
Thursday, July 29, 2010
"It's a horrible disease reallyThese were the words of Ballymahon native Tom Colohan when he was explaining the condition he suffers from - Friedrich's AtaxiaA rare genetic condition, Friedrich's Ataxia is present from birth but doesn't develop until later in life. It generally develops between t
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Glendive Girl Represents MT in Washington, DC
Friday, June 11, 2010
Billings -- A Glendive girl with a rare genetic disease is representing Montana in Washington DCAbryanna Nelson, 11, was treated at Shodair Children's Hospital in Helena for an inherited disease called Friedreich's ataxia. Shodair is one of 170 Children's Miracle Network hospitals in the country
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Family of Local Boy Raises Money to Benefit Others
Tuesday, May 25, 2010
As a young child, Sam Brown's mother said the family never suspected anything was seriously wrong with their childHis frequent falls were attributed to him being "a little on the clumsy side," she said. However, as he grew older and his gait problems became more noticeable Lee Anna Brown and her h
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Repligen Receives Orphan Drug Designation From the FDA for RG2833 for Friedreich's Ataxia
Tuesday, May 25, 2010
WALTHAM, Mass. -- Repligen Corporation (Nasdaq: RGEN) announced today that the Office of Orphan Products Development of the Food and Drug Administration (FDA) has granted orphan drug designation to RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor for the treatment of Friedreich's ataxia
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Repligen Files Investigational New Drug Application With FDA for First Drug Targeting the Core Genetic Defect of Friedreich's Ataxia
Monday, May 17, 2010
WALTHAM, Mass. -- Repligen Corporation (Nasdaq: RGEN) announced today that it has filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitorThis is a double-blind, single ascend
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Brave Sam Travels to USA for Treatment of Crippling Illness
Thursday, May 6, 2010
A Dublin family are travelling to America in the hopes that a specialist will assist their young son who has a crippling diseaseSam McMahon (8) suffers from a rare disorder of the nervous system called Friedreich's AtaxiaThe Knocklyon schoolboy is affected by clumsiness, has difficulties with bal
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Mechanism Elucidated for a Rare Disease
Tuesday, May 4, 2010
Scientists at the Friedrich Miescher Institute for Biomedical Research (FMI, part of the Novartis Research Foundation) have dissected one of the molecular mechanisms underlying Friedreich's ataxiaIn doing so, they have shed new light on the pathogenic mechanism of the disease. These findings could
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PhRMA Honors Patient Advocates Ron and Raychel Bartek
Friday, March 19, 2010
WASHINGTON -- Pharmaceutical Research and Manufacturers of America (PhRMA) President and CEO Billy Tauzin today honored Ron and Raychel Bartek for their dedicated work driving awareness of Friedreich's ataxia, a debilitating, life-threatening, rare diseaseThe Barteks are co-founders of the Friedre
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Disabled St. Albans Schoolboy Skydives for Charity
Thursday, February 18, 2010
A thrill-seeking disabled teenager living with a rare genetic disorder will fulfill a life-long dream by leaping out of a plane in aid of a charity close to his heartGlen Shorey, 16, of Corinium Gate, St Albans, has been left wheelchair-bound by his condition, medically dubbed Freidreich's ataxia,
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Excess DNA Damage Found in Cells of Patients With Friedreich's Ataxia
Friday, January 15, 2010
PITTSBURGH -- Elevated levels of DNA damage have for the first time been found in the cellular mitochondria and nuclei of patients with the inherited, progressive nervous system disease called Friedreich's ataxia (FRDA), says a multicenter research team led by an expert from the University of The
