Ataxia News and Research

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Ataxia is a neurological sign and symptom that consists of gross lack of coordination of muscle movements. Ataxia is a non-specific clinical manifestation implying disfunction of the parts of the nervous system that coordinate movement, such as the cerebellum.

FDA grants Fast Track designation to Edison’s lead drug EPI-743

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.

17 Mar 2014

Edison Pharmaceuticals initiates Phase 2B/3 study of EPI-743 in children with Leigh syndrome

Edison Pharmaceuticals today announced the initiation of a phase 2B/3 study entitled, "A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome" to be conducted in conjunction with Dainippon Sumitomo Pharma Co, Ltd.

10 Mar 2014

Longtime allies team up to create Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

Three longtime allies have joined forces to create the new Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence.

7 Mar 2014

UMMS launches Voyager Therapeutics to develop life-changing treatments for fatal diseases

UMass Medical School (UMMS) recognizes the launch today of Voyager Therapeutics, a gene therapy company founded by four world leaders in the fields of AAV gene therapy, RNA biology and neuroscience, to develop life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS).

13 Feb 2014

Researchers find link between mutations in gene called RAB 24 and inherited neurodegenerative disease

Researchers from North Carolina State University have found a link between a mutation in a gene called RAB 24 and an inherited neurodegenerative disease in Old English sheepdogs and Gordon setters. The findings may help further understanding of neurodegenerative diseases and identify new treatments for both canine and human sufferers.

7 Feb 2014

Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

Edison Pharmaceuticals today announced that the US Food and Drug Administration has granted Orphan Status to vatiquinone for the treatment of Friedreich's ataxia.

4 Feb 2014

FARA funds gene therapy-based research project to tackle Friedreich's ataxia

The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine, in Barcelona, and the "Centro de Biolog-a Molecular Severo Ochoa", in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance.

29 Jan 2014

Merck and CNIO sign agreement to collaborate in area of cancer drug development

The Spanish National Cancer Research Centre (CNIO) and the German chemical and pharmaceutical company Merck today signed an agreement in Madrid to collaborate in the area of cancer drug development.

19 Dec 2013

Merck signs license agreement with CNIO for cancer drug development

Merck Serono, the biopharmaceutical division of Merck, today announced that a license agreement has been signed with the Spanish National Cancer Research Centre in Madrid in the area of cancer drug development. The global agreement will build upon CNIO research discoveries to encourage the development and commercialization of new compounds in the field of oncology.

18 Dec 2013

Eisai reports new data analyses on FYCOMPA (Perampanel) at AES meeting

Today, Eisai Inc. announced that 16 abstracts highlighting new data analyses on FYCOMPA (perampanel) will be presented at the 67th annual American Epilepsy Society (AES) meeting, taking place in Washington, D.C. from December 6 – 10.

6 Dec 2013

Scientists join patients to address Friedreich's Ataxia

The Spanish Federation of Ataxia (FEDAES)—in representation of the GENEFA Platform for a Friedreich's Ataxia cure—, the Babel Family association for biomedical research into Friedreich's Ataxia, the "Centro de Biología Molecular Severo Ochoa" (CMBSO), and the Institute for Research in Biomedicine (IRB Barcelona) have signed an agreement through which these patients' associations will fund, by means of donations, a 3-year research project addressing Friedreich's Ataxia.

16 Nov 2013

Viropharma announces financial results for third quarter 2013

ViroPharma Incorporated reported today its financial results for the third quarter ended September 30, 2013. Net sales were $113 million for the third quarter ended September 30, 2013, as compared to $91 million in the same three months of 2012.

31 Oct 2013

Researchers identify likely molecular pathway that causes untreatable neurodegenerative diseases

University of Adelaide researchers have identified a likely molecular pathway that causes a group of untreatable neurodegenerative diseases, including Huntington's disease and Lou Gehrig's disease.

19 Sep 2013

Study shows link between cerebellum and body's ability to sense movement and limb position

Researchers at the Kennedy Krieger Institute announced today study findings showing, for the first time, the link between the brain's cerebellum and proprioception, or the body's ability to sense movement and joint and limb position.

5 Sep 2013

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

Stem cell-based epilepsy approach may help in search for better medicines to treat seizure disorders

A new stem cell-based approach to studying epilepsy has yielded a surprising discovery about what causes one form of the disease, and may help in the search for better medicines to treat all kinds of seizure disorders.

25 Jul 2013

Specific types of intestinal bacteria are major contributors to lymphoma

Researchers from UCLA's Jonsson Comprehensive Cancer Center (JCCC) have discovered that specific types of bacteria that live in the gut are major contributors to lymphoma, a cancer of the white blood cells that are part of the human immune system.

16 Jul 2013

Human gene expression mechanism opens new therapeutic strategies against neurological disease

In a study that could change the way scientists view the process of protein production in humans, University of Chicago researchers have found a single gene that encodes two separate proteins from the same sequence of messenger RNA.

4 Jul 2013

Concert Pharmaceuticals announces initiation of CTP-354 Phase 1 trial in healthy volunteers

Concert Pharmaceuticals, Inc. today announced that it has initiated a Phase 1 clinical trial with CTP-354. CTP-354 is a novel GABAA receptor subtype-selective modulator that has demonstrated no sedation at therapeutic doses in preclinical models in contrast to existing GABAA receptor non-selective agonists, such as benzodiazepines.

10 Jun 2013

Study could open up new possibilities for early diagnosis of motor disorders

The scientists report these findings in the current online edition of "The Lancet Neurology". This pan-European study could open up new possibilities of early diagnosis and smooth the way for treatments which tackle diseases before the patient's nervous system is irreparably damaged.

24 May 2013