Friedreich's Ataxia News and Research

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Friedreich's ataxia is an inherited disease that causes progressive damage to the nervous system resulting in symptoms ranging from gait disturbance and speech problems to heart disease. A well-known celebrity afflicted with Friedreich's ataxia is former major leaguer and noted historian Clinton Stewart.

Viropharma announces financial results for second quarter of 2013

VIROPHARMA INCORPORATED (Nasdaq: VPHM) today announced financial results for the second quarter of 2013. Net sales were $104 million for the second quarter ended June 30, 2013 as compared to $95 million in the comparative period of 2012.

1 Aug 2013

ViroPharma announces results of VP20621 Phase 2 study for preventing recurrent CDI

ViroPharma Incorporated, an international biopharmaceutical company committed to developing and commercializing innovative products that address unmet medical needs and rare diseases, today announced the results of a Phase 2 study of VP20621 (non-toxigenic Clostridium difficile; NTCD) a novel treatment approach for preventing recurrent C. difficile infections.

23 Apr 2013

Friedreich's Ataxia costs between £11,000 and £19,000 per person per year

What is the cost of rare diseases such as Friedreich's Ataxia? By analyzing direct and indirect costs of care research in BioMed Central's open access journal Orphanet Journal of Rare Diseases calculated that conservatively this rare disease costs between £11,000 and £19,000 per person per year.

28 Feb 2013

Children's Oakland recruits patients for deferiprone clinical trial to treat PKAN

A groundbreaking, international clinical trial of an iron chelation drug, deferiprone, to treat the rare and devastating neurodegenerative disease, pantothenate kinase-associated neurodegeneration (PKAN), began last month at the trial's only North American site, Children's Hospital & Research Center Oakland.

17 Jan 2013

Edison Pharmaceuticals to commence EPI-743 phase 2B Friedreich's ataxia study

Edison Pharmaceuticals today announced the initiation of a phase 2B study entitled, "Safety and Efficacy Study of EPI-743 on Visual Function in Patients with Friedreich's Ataxia."

7 Jan 2013

Study discovers pathways that govern instability of GAA/TTC repeats

A study of more than 6,000 genes in a common species of yeast has identified the pathways that govern the instability of GAA/TTC repeats. In humans, the expansions of these repeats is known to inactivate a gene - FXN - which leads to Friedreich's ataxia, a neurodegenerative disease that is currently incurable. In yeast, long repeats also destabilize the genome, manifested by the breakage of chromosomes.

11 Sep 2012

Friedreich Ataxia Research Alliance, USF ARC to host ‘Understanding a Cure’ symposium

Scientists, clinicians and patients will gather at the University of South Florida's Center for Advanced Medical Learning and Simulation (CAMLS) in downtown Tampa Sept. 6 to share research insights and energize the search for a cure for Friedreich's ataxia and related disorders.

28 Aug 2012

ViroPharma initiates maribavir Phase 2 program for CMV infections

ViroPharma Incorporated today announced the initiation of a Phase 2 program to evaluate maribavir for the treatment of cytomegalovirus infections in transplant recipients.

4 Jun 2012

Intellect Neurosciences announces two new programs in Alzheimer's disease development pipeline

Intellect Neurosciences, Inc., a biopharmaceutical company engaged in the discovery and development of disease-modifying therapeutic agents for the treatment of Alzheimer's and other neurological diseases, today announced two new programs in its Alzheimer's disease development pipeline. The new programs are based on two antibodies that target early neurotoxic forms of tau protein, which Intellect intends to develop for therapeutic and diagnostic uses.

7 May 2012

Repligen total revenue increases to $7,165,000 for quarter ended December 31, 2011

Repligen Corporation today reported financial results for the quarter and nine-month fiscal year ended December 31, 2011.

17 Mar 2012

Intellect Neurosciences receives U.S. patent allowance for ANTISENILIN to treat AD

Intellect Neurosciences, Inc., a biopharmaceutical company engaged in the discovery and development of disease-modifying therapeutic agents for the treatment of Alzheimer's and other neurological diseases announced today that it received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for patent claims covering the company's ANTISENILIN monoclonal antibody platform technology for the treatment and prevention of Alzheimer's disease.

7 Mar 2012

ViroPharma's NDS for Cinryze receives Health Canada's Priority Review status

ViroPharma Incorporated today announced that Health Canada has granted the company Priority Review status for its New Drug Submission (NDS) for Cinryze (C1 inhibitor [human]).

13 Jan 2012

ViroPharma to acquire Meritage

ViroPharma Incorporated today announced that it has entered into an exclusive option to acquire Meritage Pharma, Inc., a private company based in San Diego, CA focused on developing oral budesonide suspension (OBS) as a treatment for eosinophilic esophagitis (EoE), a chronic inflammatory disorder of the esophagus.

23 Dec 2011

UPC develops active knee-ankle orthosis to assist gait in people with incomplete SCI

A team from the Department of Mechanical Engineering and the Biomedical Engineering Research Centre (CREB) of the Universitat Politècnica de Catalunya. BarcelonaTech (UPC) has developed an active knee-ankle orthosis to assist gait in people with incomplete spinal cord injury. The project is being carried out in collaboration with the University of A Coruña and the University of Extremadura.

12 Oct 2011

FARA announces 2011 Ataxian Athlete Initiative grant recipients

The Friedreich's Ataxia Research Alliance (FARA), in partnership with The FA Project, Catrike, Anna Maria Oyster Bar, and The Melting Pot are pleased to announce Bill Ramsey, Tom Trovinger, Mary Vida, and Patrick Cogan as the 2011 Ataxian Athlete Initiative (AAI) grant recipients.

10 Oct 2011

ViroPharma licenses worldwide rights from Intellect Neurosciences to OX1 for treatment of FA

ViroPharma Incorporated today announced the license of worldwide rights from Intellect Neurosciences, Inc. to its clinical stage drug candidate, OX1, being developed for the treatment of Friedreich's Ataxia (FA), a rare, hereditary, progressive neurodegenerative disease.

1 Oct 2011

MDA announces $13.7M in grants to 40 new muscular dystrophy research initiatives

The Muscular Dystrophy Association today announced funding, totaling $13.7 million, for 40 new research initiatives targeting nearly two dozen progressive neuromuscular diseases.

24 Aug 2011

Repligen first quarter total revenue increases 9% to $7,654,000

Repligen Corporation today reported results for the first quarter of fiscal year 2012, ended June 30, 2011.

27 Jul 2011

Preliminary results from Edison's EPI-A0001 28-day phase 2A trial against Friedreich's ataxia

Edison Pharmaceuticals, Inc. announced today preliminary results obtained on a 28-day phase 2A clinical trial in Friedreich's ataxia. While the primary endpoint of glucose disposition index did not show statistically significant improvement, EPI-A0001 did significantly improve neurological function as assessed by the Friedreich's Ataxia Rating Scale.

10 Jun 2011

Repligen total revenue increases 30% to $27,291,000 for fiscal year 2011

Repligen Corporation today reported results for the fourth quarter and fiscal year 2011, ended March 31, 2011.

9 Jun 2011