Heart transplantation could be a viable option in the long-term for Friedreich ataxia (FRDA) patients as suggested by the cases of three patients who remained alive, cognitively intact, and socially engaged following heart transplantation.
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A cocktail of clinically approved cytokines for other diseases shows promising neuro-protective effects in a Friedreich’s ataxia (FA) humanized mouse model. The study, “Cytokine therapy-mediated neuroprotection in a Friedreich’s ataxia mouse model,” was published in the journal Annals of Neurology. FA is a severe neurological disease that affects the…
American researchers have come up with a new, non-invasive way to measure mitochondrial function in human skeletal muscle, a development that could help those with Friedreich’s ataxia (FA) and other diseases. Aberrant functioning of mitochondria, cell components that convert food to energy, is a hallmark of such disorders. The team from Ohio State…
Researchers have successfully corrected a mutated frataxin gene that leads to reduced production of the frataxin (FTX) protein and development of disease in mice, according to new research. This achievement provides new insight into laboratory methods to study the disease and supports the therapeutic potential of gene editing techniques to…
The most obvious neurological symptoms of Friedreich’s ataxia are incoordination of movement, gait disturbances and dizziness confirms a study published in journal International Archives of Otorhinolaryngology. Changes in the vestibular or balance system also occur in the vast majority of patients. Conducing vestibular testing is therefore important since otoneurologic symptoms, or those associated with the portions of the nervous system related to the ear occur early and this information could help doctors decide which procedure to perform when treating patients with Friedreich’s ataxia.
Patients with different muscular-coordination conditions, or ataxia, exhibit different eye reflexes after head impulses, a finding that could help doctors determine the severity of a patient’s disease. The video head impulse tests (vHIT) that researchers used showed differences in eye movements between patients with spinocerebellar ataxias and patients with Friedreich’s…
Fibroblasts, or connective tissue cells, from Friedreich’s ataxia patients were used to identify changes in the frataxin (FXN) gene under stress, and to see how it interacts in various regulatory and biological processes. This basic genetics research, in helping to explain and understand the disease, may help to guide the development of…
The Friedreich’s Ataxia Research Alliance (FARA) and Livonia, Michigan-based law firm Foley, Baron, Metzger & Juip (FBMJ) have teamed up to sponsor a movie premiere event featuring the highly anticipated “Rogue One: A Star Wars Story.” Proceeds will be designated to support research toward finding treatments and…
Horizon Pharma has dropped its entire Actimmune (interferon gamma-1b) clinical development program in Friedreich’s ataxia following the failure of a Phase 3 trial which aimed to show that the treatment improves outcomes of the disease. The results are disappointing for patients with a disease lacking any effective treatment. The STEADFAST…
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Altered Cellular Metabolism in Friedreich’s Ataxia Is Potential Therapeutic Target, Review Says
Recent research in Friedreich’s ataxia (FA) has revealed new possibilities for lipid metabolism as a therapeutic target to slow the progression of the disease in patients. The latest knowledge of the molecular basis of FA and its underlying metabolic alterations was discussed in a review study titled “Oxidative stress…
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