Elamipretide is a peptide compound developed by Stealth Therapeutics. It targets the inner mitochondrial membrane where it binds reversibly to

PTC-743 / Vatiquinone (previously EPI-743), is a follow-on or “sister” compound to EPI-A0001, which aims to improve mitochondrial and cellular

It is important to understand the burden of F A, including its extent and the most salient elements. The objective

Omaveloxolone, also called omav, is one of a class of drugs that Reata has developed to target the activation of

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