News

Cerebellar Changes in Ataxias Detected by fMRI

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…

Inhibiting Iron Transport Improves FA in Fruit Flies

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…

BioBlast Pharma Announces Promising Preclinical Results on a New Therapy for Friedreich’s Ataxia

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…

HDAC Inhibitors Studied as Novel Friedreich’s Ataxia Treatment

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…