
Mitochondrial medicine therapies: rationale, evidence, and dosing guidelines
Author(s) -
Isabella Peixoto de Barcelos,
Edward Charles Shadiack,
Rebecca Ganetzky,
Marni J. Falk
Publication year - 2020
Publication title -
current opinion in pediatrics, with evaluated medline/current opinion in pediatrics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.817
H-Index - 87
eISSN - 1080-8116
pISSN - 1040-8703
DOI - 10.1097/mop.0000000000000954
Subject(s) - medicine , mitochondrial disease , intensive care medicine , dosing , lactic acidosis , disease , bioinformatics , pharmacology , pathology , biology , biochemistry , gene , mitochondrial dna
Primary mitochondrial disease is a highly heterogeneous but collectively common inherited metabolic disorder, affecting at least one in 4300 individuals. Therapeutic management of mitochondrial disease typically involves empiric prescription of enzymatic cofactors, antioxidants, and amino acid and other nutrient supplements, based on biochemical reasoning, historical experience, and consensus expert opinion. As the field continues to rapidly advance, we review here the preclinical and clinical evidence, and specific dosing guidelines, for common mitochondrial medicine therapies to guide practitioners in their prescribing practices.