News

Catabasis Pharmaceuticals, Inc., recently announced that it will receive the Kyle Bryant Translational Research Award, given by the Friedreich’s Ataxia Research Alliance (FARA), to further evaluate CAT-4001, a potential Friedreich’s ataxia (FA) drug. CAT-4001 is being developed by Catabasis as a potential treatment for neurodegenerative diseases, such as FA and amyotrophic lateral…

Corticosteroid therapy is a potential therapeutic treatment for patients with Friedreich ataxia (FRDA) and nephrotic syndrome, according to a study titled “Friedreich Ataxia and nephrotic syndrome: a series of two patients,” published in the journal BMC Neurology. FRDA is a genetic, progressive, neurodegenerative disorder that causes…

Bamboo Therapeutics, Inc., a biotechnology company focused on developing gene therapies for rare central nervous system and neuromuscular diseases, including Friedrich’s ataxia, announced that it has acquired the University of North Carolina (UNC) at Chapel Hill’s viral Vector Core gene therapy manufacturing facility. Financial details of the deal…

Annapurna Therapeutics has just announced it will collaborate with Weill Cornell Medicine to develop a market-leading partnership specialized in gene therapy. One of these therapies currently under Annapurna’s exclusive development targets cardiomyopathy as it is associated with Friedreich’s ataxia, a disease that affects between 10,000 and 20,000 people in the U.S. and Europe. Friedreich’s ataxia…

Pfizer has published a study with potentially important implications for future therapeutic targets to treat Friedreich’s ataxia (FA). The study, titled “Perturbation of cellular proteostasis networks identifies pathways that modulate precursor and intermediate but not mature levels of frataxin,” was conducted through Pfizer’s Rare Disease Research…

A recent study links frataxin protein levels to disease features in patients with Friedreich’s ataxia (FA). The article, titled “Frataxin levels in peripheral tissue in Friedreich ataxia,” was published in Annals of Clinical and Translational Neurology journal. FA is a neurological disorder that progressively damages the nervous…

Researchers have long known that — due to a genetic mutation —  the levels of the protein frataxin are decreased in Friedreich’s ataxia. Because of the neurological nature of the disease, the effects of the mutation have mainly been studied in neurons within the central nervous system, and not much is known about…

New research indicates that a complete assessment of heart function can identify cardiomyopathy in most people with Friedreich’s ataxia (FA), and that electrocardiography, especially, shows early heart abnormalities. The work, titled “The cardiomyopathy in Friedreich’s ataxia — New biomarker for staging cardiac involvement,” appeared in the…

Researchers have shown that a potential new therapeutic target for patients with Friedreich’s ataxia (FA) could be a mutation that causes misfolding in specific proteins, which results in lowered protein concentrations. The study, led by senior scientists at the Department of Basic and Clinical Neuroscience, Maurice Wohl Institute, King’s…

In a new study entitled “Efficient attenuation of Friedreich’s ataxia (FRDA) cardiomyopathy by modulation of iron homeostasis-human induced pluripotent stem cell (hiPSC) as a drug screening platform for FRDA,” researchers suggest that the chemical compound deferiprone, a mitochondrial permeable iron chelator, is an effective drug to treat Friedreich’s ataxia-mediated cardiomyopathy by…