Mitochondrial Disease

Mitochondrial disease causes, types, symptoms, prognosis & treatment

Newly Discovered Mitochondrial Mutation in the MOTS-c Region Increases the Risk of Diabetes in Japanese Men

One year ago, the LifeArc Centre for Rare Mitochondrial Diseases was created to help tackle some of the biggest challenges in mitochondrial disease. 🧪 Slow diagnosis 📉 Difficulties tracking how the condition changes over time 🧫 Lack of tests to help show whether a potential treatment is working ❌ No cures We’re proud to support this centre, which brings together experts and people affected by these conditions to improve diagnosis and speed up the search for new treatments. 🎥 Hear from the re

The @fda just denied the ONLY treatment option for my daughter’s rare, life-limiting mitochondrial disease (PDCD). DCA is a well understood drug with a strong safety profile and my daughter has been on it for over 3 years. Losing access to DCA will have catastrophic effects on her quality of life. With such a terrible disease as PDCD, a drug that could even give her a relative decrease in daily pain and a few less hospital admissions each year would be worth it, but DCA has done that and so much

Recently, @FDA denied DCA, the ONLY treatment for my daughter’s rare, life limiting mitochondrial disease, PDCD. It was denied not because it was unsafe, but because they did not see the benefit. Please follow along over the next few weeks as I show the @FDA that quality of life benefits for a kid with a rare disease can’t always be quantified. When you live them, when each day is met with more smiles than pain, when milestones are reached that you never thought possible. That’s when you know. D