Shortcast: Use of Elamipretide in patients assigned treatment in the compassionate use program

Dr Mary Kay Koenig describes the use of elamipretide in three children with different mitochondrial disorders. Her group's work provides dosing parameters for the use of elamipretide in patients <12 years of age with mitochondrial diseases caused by pathogenic variants that impair membrane phospholipid remodelling. Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases Mary Kay Koenig, et al https://doi.org/10.1002/jmd2.12335