Reata Pharmaceuticals announced promising results from the first part of their trial. This was designed to show safety and test what dose of the medication was best to use for their next efficacy trial. They are now planning part 2 of the trial, a placebo controlled trial with around 100 people. A representative from Reata pharmaceuticals will be speaking at Ataxia UK’s annual conference. For more information see press release.
Two recent publications on a promising work using stem cells in Friedreich’s ataxia
Researchers based in Bristol (UK), who have been working on stem cells as potential therapy for Friedeich’s ataxia, have recently published two articles about their study. Ataxia UK have contributed to this research and is very pleased to see positive results being published. You can find the two publications here: Cytokine therapy-mediated neuroprotection in a Friedreich’s ataxia mouse model in Annals of Neurology and Mesenchymal stem cell-derived factors restore function to Human frataxin-deficient cells in Cerebellum.