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K.PetersonB
D.CorreiaR
In: Fogarty W.M., Kelly C.T., editors
436 Friedreichs ataxia is characterized by alterations in lipid metabolism, resulting in the accumulation of intracellular lipids in the form of lipid droplets in fibroblasts from patients
Further doseresponse and steady-state administration studies will be required to optimize the dosing schedule for future trials to evaluate therapeutic efficacy
new models should be systematically validated
