The generation of effective FRDA disease models is crucial for answering questions regarding the pathophysiology of this disease. There have

Frataxin functions in the biogenesis of iron-sulfur clusters, which are prosthetic groups that are found in proteins involved in many

Many groups worked on the creation of FRDA mouse models by decreasing the mouse frataxin or knocking it out, or

Larimar Therapeutics Announces FDA has Partially Lifted the Clinical Hold and Plans for a Phase 2 Dose Exploration Trial of

Yes. Small studies showed that it helped thin the heart walls by a small amount, with minimal, if any, improvement

The thing that I would ask most of all is that all people with FA, Friedreich’s see a cardiologist and

For mitochondrial cardiomyopathies, including Friedreich’s, we may not have a cure, but we have multiple drugs that seem to help

A particular thing about these hearts is that, over time, they maintain good systolic function [contraction of the heart muscle].

The heart symptoms may have irregular heart rhythms, which they can feel as palpitations, as the heart beats faster or

The cardiomyopathy in Friedreich’s is typically hypertrophic cardiomyopathy. That means the walls of the heart get really thick. This thickening