Everyone has an “energy baseline” or sweet spot where we have enough energy to do the things we want to do, but having mitochondrial disease means that the body has ... Read more

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the ... Read more

Patients with Leber hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes vision loss, experienced a dramatic and mostly irreversible loss of visual acuity, according to an interim ... Read more

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. ... Read more

A new mouse model may help scientists deepen their knowledge about mitochondria, the cell compartments responsible for the production of energy in the body, as well as their understanding about ... Read more

Researchers in Spain have made a discovery that has important implications for treatment strategies to prevent mitochondrial diseases from being passed from mother to child. Specifically, they found that the ... Read more

Elamipretide (MT-131), developed by Stealth BioTherapeutics, is able to improve cardiac function in Barth syndrome patients, new data from a Phase 2/3 clinical trial show. Hilary Vernon, MD, PhD, a ... Read more