Patrícia Silva, PhD,  director of science content—

A team led by researchers at the Universidad Autónoma de Madrid in Spain recently published new data on a gene therapy for Friedreich’s ataxia based on the expression of the human frataxin gene from artificial systems. The study is entitled “Delivery of the 135 kb human…

A new study led by researchers at University of Rome ‘Tor Vergata’ in Italy recently revealed that inhibitors of the Src protein kinase can represent a potential new therapeutic strategy against Friedreich’s ataxia. The study is entitled “Src inhibitors modulate frataxin protein levels” and was published…

Researchers at Miguel Hernandez University and the University of Murcia in Spain have recently published in the journal Experimental Cell Research their results on genetic and epigenetic factors linked to Friedreich’s ataxia. The study is entitled “Novel aberrant genetic and epigenetic events…

A new study recently published in the journal Neurology revealed a potential novel therapeutic approach for Friedreich’s ataxia based on analogs of incretin, a metabolic hormone that stimulates insulin secretion. The study is entitled “Study of beta cells and neurons indicate incretin analogs as…

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…

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”…

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…

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 “…

A recent opinion article from two researchers at University of Naples Federico II, Italy, entitled “Movement disorders: Friedreich ataxia today – preparing for the final battle” and published in the journal Nature Reviews Neurology, focused on the results from a European consortium based on a…