News

Chondrial Therapeutics has licensed the investigational compound CTI-1601 for treating  Friedreich’s ataxia (FA) from Indiana University Research and Technology Corporation (IURTC) and Wake Forest University Health Sciences. The company said it has also secured $22.6 million in financing to advance the treatment. “The $22.6 million Series A financing, combined with the licensing…

Treating nerve cells derived from Friedreich’s ataxia (FA) patients with a molecule called compound 109 significantly increases the expression of the frataxin gene and protects the them from cell death induced by oxidative stress, suggests a study published in the scientific journal Human Molecular Genetics.

Liver growth factor (LGF) increased the frataxin protein and reduced oxidative stress when administered to mice with Friedreich’s ataxia (FA). This approach leads to a partial restoration of motor coordination in mice and may be a new therapeutic approach to treat FA patients. The study, “Liver Growth Factor (LGF)…

Patients with Friedreich ataxia (FA) are more likely to develop inflammatory bowel disease and growth hormone deficiency than those without FA, concludes a recent study. Researchers believe that these associations, which may be coincidental or a result of disease interaction or shared genetic features, warrant further research. The study, “Comorbid Medical Conditions…

Researchers found two chemical treatments that increased iron storage protein, called mitochondria ferritin, in cells from a Friedreich’s ataxia patient. These results suggest a potential treatment option for protecting against mitochondrial oxidative damage in these patients. The study, “Characterization of human mitochondrial ferritin promoter: identification of transcription factors and…

Noninvasive measurements of mitochondrial capacity in skeletal muscle of patients with Friedreich’s ataxia could be used to monitor disease severity and progression, according to a recent study. In contrast, the study failed to find a link between mitochondrial capacity or muscle endurance to perceptions of fatigue. The study,…

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.

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…