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The nonprofit Global Genes, an organization for patients and families fighting rare and genetic diseases, is awarding more than $100,000 in travel stipends to help hundreds of patient advocates attend 5th Annual RARE Patient Advocacy Summit  Sept. 22-23 in Huntington Beach, California. After the summit, the 2016 Tribute to Champions of…

Pfizer recently acquired the biotech Bamboo Therapeutics which develops therapies for potential treatment of patients with neuromuscular and central nervous system (CNS) rare diseases – like Friedreich’s ataxia (FA). The purchase is expected to expand Pfizer’s portfolio in gene therapy by including the advanced recombinant Adeno-Associated Virus (rAAV) vector design and production technology; and a fully…

The U.S. Food and Drug Administration (FDA) has designated AGIL-FA, a gene therapy from Agilis Biotherapeutics intended as a potential treatment of Friedreich’s ataxia (FA), an Orphan Drug, a designation that speeds its development and testing. “Having the first gene therapy product candidate to receive orphan drug designation from the FDA for…

Whether or not to participate in a clinical trial is a hard decision to make — but when it comes to deciding for a child, the task can be the hardest of a lifetime. When Sandra Sojka Lagedrost’s daughter Joanna was only 10 years old and diagnosed with Friedreich’s ataxia (FA) questions…

Researchers at Weill Cornell Medical College in New York have discovered mechanisms that explain how a mutation in the frataxin gene — the cause of Friedreich’s ataxia — prevents the production of the frataxin protein, and also discovered a process by which the mutations expand. The study, “…

Improving metal homeostasis was shown to have therapeutic benefits in a fly model of human Friedreich’s ataxia (FA). The study “Metal Homeostasis Regulators Suppress FRDA Phenotypes in a Drosophila Model of the Disease,” was published in the journal PLOS One. Friedreich’s ataxia is a neurodegenerative disease caused by reduced levels…

A new form of sun protection that works by chelating, or binding, excess iron within mitochondria may not only protect against UVA rays that current lotions are unable to block, but also lead to new treatments for  Friedreich’s ataxia (FA). The compound, named “mitoiron claw” by the scientific team in the U.K. that developed it, is thought capable…

The mechanism leading to cell death in Friedreich’s Ataxia (FRDA) has been described in a new study published in the Annals of Clinical and Translational Neurology.

Neurodegeneration in Friedreich’s ataxia can be independent of faulty mitochondria and oxidant molecules. Rather, researchers at Baylor College of Medicine in Houston, Texas, demonstrated that iron toxicity contributed to disease processes in a fruit fly model of the disease. The study, “Loss of Frataxin induces iron toxicity, sphingolipid synthesis, and…

The Friedreich’s Ataxia Research Alliance (FARA) has announced the 2016 winners of the Ataxian Athlete Initiative (AAI), a unique program providing adaptive cycling equipment to people with ataxia who have shown a strong commitment to staying healthy and fit despite their progressive illness. Since 2009, 33 people have been…