News

Researchers at the University of Oxford in the United Kingdom recently published in the journal Human Molecular Genetics new insights into the molecular mechanisms behind the repression of the frataxin gene, which is associated with Friedreich’s ataxia. The study is entitled “Expanded GAA repeats impair…

Specialty biopharmaceutical Horizon Pharma plc has been awarded Fast Track status from the U.S. Food and Drug Administration (FDA) for the company’s ACTIMMUNE (interferon gamma-1b) to be used in patients with Friedreich’s ataxia (FA). ACTIMMUNE is a biologically developed protein resembling one produced naturally by the body to prevent infection,…

A new study was recently published in the journal Parkinsonism & Related Disorders, revealing that sleep disorders can be helpful in the differentiation of different ataxia types. The study is entitled “Differential Diagnosis of Sporadic Adult-Onset Ataxia: The Role of REM Sleep Behavior Disorder”…

Patients with Friedreich’s ataxia may have more damage to brain cells than was previously thought. A study from University of Duisburg-Essen in Germany led by Dr. Maria R. Stefanescu and principal investigator Dr. Dagmar Timmann used magnetic resonance imaging (MRI) to look at the levels of atrophy in various…

A research group in Germany at University of Regensburg that considers Friedreich’s ataxia “the most important recessive ataxia in the Caucasian population” is breaking down the complex disease using genetically modified fruit flies. Appearing in the journal Free Radical Biology and Medicine, their most…

Researchers at Wayne State University in Detroit and the University of Pennsylvania in Philadelphia, recently revealed the mechanism behind the iron binding process in the assembly of iron-sulfur (Fe-S) clusters, a pathway that is defective in disorders such as Friedreich’s ataxia. The study was published in the journal…

Biotechnology company BioBlast Pharma Ltd. recently announced in a press release that its mitochondrial protein replacement therapy drug candidate (BB-FA) for Friedreich’s ataxia has yielded positive results in a preclinical in vitro and in vivo proof-of-concept study. The results were presented during the International Ataxia Research Conference recently…

Inhibitors of 2-aminobenzamide histone deacetylase (HDAC) are a proposed new treatment for Friedreich’s ataxia. Applying HDAC inhibitors to neuronal cells derived from Friedreich’s ataxia patients’ induced-pluriopotent stem cells results in an increased expression of frataxin mRNA transcripts and protein. A group of researchers at The Scripps Research Institute in…

In a recent article titled “Consensus clinical management guidelines for Friedreich ataxia” published in the journal Orphanet Journal of Rare Diseases, Louise Corben from the Children’s Research Institute, Australia and colleagues on behalf of the Clinical Management Guidelines Writing Group Murdoch reviewed the…

Researchers at the University of Rome “Tor Vergata” in Italy and Fratagene Therapeutics Ltd. in Ireland recently revealed a novel therapeutic strategy for Friedreich’s ataxia based on specific small molecules. The study was published in the journal Neurobiology of Disease and is entitled “…