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Last updated on April 23, 2014 at 10:37 EDT

Latest Nikolaus Friedreich Stories

2014-04-07 16:08:27

The transfer, via a viral vector, of a normal copy of the gene deficient in patients, allowed to fully and very rapidly cure the heart disease in mice. These findings are published in Nature Medicine on 6 April, 2014. Friedreich's ataxia is a severe, rare hereditary disorder which combines progressive neuro-degeneration, impaired heart function and an increased risk of diabetes. The condition affects one in every 50,000 birth. There is currently no effective treatment for this disease. In...

2014-04-06 16:20:26

AAVLife aimed at advancing gene therapy for cardiomyopathy in Friedreich's ataxia to clinic DOWNINGTOWN, Pa., April 6, 2014 /PRNewswire-USNewswire/ -- The Friedreich's Ataxia Research Alliance (FARA) is pleased to recognize the ground-breaking gene-therapy research of FARA-funded scientist Dr. Hélène Puccio. In the current online issue of Nature Medicine, Dr. Puccio and her colleagues present significant results demonstrating that gene-replacement therapy using an adeno-associated...

2014-03-19 23:25:02

EPI-743 gains key regulatory endorsement. Downingtown, PA, (PRWEB) March 19, 2014 Edison Pharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Status to EPI-743 for the treatment of Friedreich’s ataxia (http://www.prnewswire.com/news-releases/fda-awards-fast-track-status-to-edison-pharmaceuticals-epi-743-for-friedreichs-ataxia-250558901.html). This status will further accelerate the clinical development of EPI-743 now under way in two...

2014-03-17 00:23:45

EPI-743 in two phase 2 development studies for Friedreich's ataxia MOUNTAIN VIEW, Calif., March 17, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the United States Food and Drug Administration granted Fast Track designation to EPI-743, the company's lead drug, for the treatment of Friedreich's ataxia. EPI-743 is being developed for pediatric and adult mitochondrial disease, including Friedreich's ataxia. EPI-743 has successfully completed phase 1 and multiple...

2014-02-05 23:23:28

Repligen Corporation announced that BioMarin Pharmaceutical has acquired the assets in the company’s Histone DeACetylase (HDAC) inhibitor program. Downingtown, PA (PRWEB) February 05, 2014 Repligen Corporation announced that BioMarin Pharmaceutical has acquired the assets in the company’s Histone DeACetylase (HDAC) inhibitor program. Repligen has been advancing a therapeutic research and development program in Friedreich's Ataxia (FA) in which specific HDAC inhibitors have been...

2014-02-04 23:00:35

EPI-743 phase 2b Friedreich's ataxia trial fully enrolled. Downingtown, PA (PRWEB) February 04, 2014 Edison Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to vatiquinone (EPI-743) for the treatment of Friedreich’s ataxia (http://www.prnewswire.com/news-releases/fda-grants-edison-pharmaceuticals-epi-743-orphan-status-for-friedreichs-ataxia-243439351.html). Orphan status brings additional momentum and acceleration to the...

2014-02-04 00:21:05

EPI-743 in phase 2 development for Friedreich's ataxia MOUNTAIN VIEW, Calif., Feb. 4, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the US Food and Drug Administration has granted Orphan Status to vatiquinone for the treatment of Friedreich's ataxia. Vatiquinone is the International Nonproprietary Name (INN) for Edison's EPI-743. The INN is a unique international name issued by the World Health Organization. It is used to identify the active pharmacological...

2013-12-19 12:29:09

Girls with Rare Disease (Friedreich's Ataxia) Selected as 'Safe Step Cares' Honorees INDIANAPOLIS, Dec. 19, 2013 /PRNewswire/ -- It began with a letter to a company in Nashville about a teenage girl in Indianapolis with a rare, life threatening disease. Nathan Hand, a friend of the girl's family, wrote to Safe Step Walk-In Tub Co., which had recently donated a state-of-the art therapeutic tub through its nationwide program of giving, Safe Step Cares. The recipient, Selena Yorke,...

2013-11-04 00:21:27

First patient enrolled to evaluate efficacy in rare Friedreich's ataxia genotype MOUNTAIN VIEW, Calif. and DOWNINGTOWN, Pa. and TAMPA, Fla., Nov. 4, 2013 /PRNewswire/ -- Edison Pharmaceuticals, the Friedreich's Ataxia Research Alliance (FARA) and the University of South Florida (USF) today announced the initiation of a phase 2 study entitled, "Phase 2A Clinical Trial of EPI-743 on Visual Function in Friedreich's Ataxia Patients with Point Mutations." Given the rarity of the...

2013-02-28 14:16:28

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. Proper understanding resource allocation is important in minimizing the effect of Friedreich's Ataxia on people's lives while maximizing their quality of life. Diseases are classified...