Friedreich's Ataxia News and Research RSS Feed - Friedreich's Ataxia News and Research

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.
Edison's EPI-743 receives FDA orphan drug designation for treatment of Leigh syndrome

Edison's EPI-743 receives FDA orphan drug designation for treatment of Leigh syndrome

Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome. [More]
Research suggests that person can slow aging process by exercising regularly

Research suggests that person can slow aging process by exercising regularly

New research by Canadian sports medicine physician Mark Tarnopolsky, MD, PhD, suggests that a person can slow the speed at which they age by exercising regularly. Dr. Tarnopolsky presented his research titled, "Exercise as a Countermeasure for Aging: From Mice to Humans" today at the 23rd Annual Meeting of the American Medical Society for Sports Medicine (AMSSM). Dr. Tarnopolsky discussed how regular exercise not only improves the quality of life but can also extend a person's lifespan by up to five years. [More]
Edison Pharmaceuticals begins EPI-743 clinical trial in children with Pearson syndrome

Edison Pharmaceuticals begins EPI-743 clinical trial in children with Pearson syndrome

Edison Pharmaceuticals today announced the initiation of a phase 2 study entitled "A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone™) in Children with Pearson Syndrome." The Investigative New Drug application (IND) was approved by the Food and Drug Administration, Office of Hematology and Oncology Products. [More]
FDA grants Fast Track designation to Edison’s lead drug EPI-743

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

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

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. [More]
UMMS launches Voyager Therapeutics to develop life-changing treatments for fatal diseases

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). [More]
Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

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. [More]
FARA funds gene therapy-based research project to tackle Friedreich's ataxia

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. [More]
Scientists join patients to address Friedreich's Ataxia

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. [More]
Viropharma announces financial results for third quarter 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. [More]
Viropharma announces financial results for second quarter of 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. [More]
ViroPharma announces results of VP20621 Phase 2 study for preventing recurrent CDI

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. [More]
Friedreich's Ataxia costs between £11,000 and £19,000 per person per year

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. [More]
Children's Oakland recruits patients for deferiprone clinical trial to treat PKAN

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. [More]
Edison Pharmaceuticals to commence EPI-743 phase 2B Friedreich's ataxia study

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." [More]
Study discovers pathways that govern instability of GAA/TTC repeats

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. [More]
Friedreich Ataxia Research Alliance, USF ARC to host ‘Understanding a Cure’ symposium

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. [More]
ViroPharma initiates maribavir Phase 2 program for CMV infections

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. [More]
Intellect Neurosciences announces two new programs in Alzheimer's disease development pipeline

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. [More]