News

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…

Rating scales used to succinctly describe the severity of Friedreich’s ataxia and the impact on patients’ quality of life have a tradeoff between being comprehensive enough to adequately describe patient symptoms and overly detailed and complicated. A new study published in Journal of the Neurological Sciences…

Patients with Friedreich’s ataxia have genetic mutations that lead to frataxin protein deficiency, but when their cells are treated in vitro with a class of molecules called Src inhibitors, frataxin protein accumulates. This finding was the result of a series of experiments described in the article, “…

The Research Acceleration and Innovation Network (TRAIN) has invited nonprofit organization Friedreich’s Ataxia Research Alliance (FARA), which works nationwide to support research and find a cure for the rare neuromuscular disease Friedreich’s ataxia (FA), to join its network of organizations. The program was launched in 2005 by FasterCures and…

A long string of DNA repeats are the molecular basis for Friedreich’s ataxia. The beginning of the first intron of FXN, the gene encoding the protein frataxin, has an abnormally large number of GAA repeats in Friedreich’s ataxia patients compared to normal individuals. A large body of evidence shows that…

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…

It is well known that a DNA mutation causes Friedreich’s ataxia by reducing the production of frataxin protein. Patients with Friedreich’s ataxia often have several hundred “GAA repeats” in their DNA, whereas unaffected individuals have no more than 40 GAA repeats. What is less known is how exactly GAA repeats…

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…

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,…