How does nomlafobusp help with FA?

José Lopes, PhD avatar

by José Lopes, PhD |

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Category: CTI-1601

Nomlafobusp is an experimental therapy that is not yet approved for the treatment of Friedreich’s ataxia. It is designed to deliver a functional version of frataxin — a protein whose shortage causes Friedreich’s ataxia — to help improve the functionality of mitochondria, the so-called “powerhouses” of cells. This is expected to help ease FA symptoms.