Therapeutic Prospects for Friedreich’s Ataxia

Publication date: April 2019Source: Trends in Pharmacological Sciences, Volume 40, Issue 4Author(s): Siyuan Zhang, Marek Napierala, Jill S. NapieralaFriedreich’s ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.
Source: Trends in Pharmacological Sciences - Category: Drugs & Pharmacology Source Type: research