|Åsander Frostner E, Simón Serrano S, Chamkha I, Donnelly E, Elmér E, Hansson MJ (2022) Towards a treatment for mitochondrial disease: current compounds in clinical development. https://doi.org/10.26124/mitofit:2022-0014 - 2022-06-28 Published in »Bioenergetics Communications 2022.4«|
» MitoFit Preprints 2022.14.
Abstract: Aasander Frostner 2022 Abstract Bioblast: Primary mitochondrial diseases are a heterogeneous group of rare genetic disorders affecting approximately 125 persons per million. Mutations underlying these diseases give rise to biological changes (including decrease in energy production and increase in reactive oxygen species), leading to organ failure, and commonly early morbidity. Mitochondrial diseases often present in early childhood and lead to the development of severe symptoms, with severe fatigue and myopathy being some of the most prevalent and debilitating ones.
There is currently no cure for primary mitochondrial diseases, nor any approved pharmaceutical treatments for multisystemic disorders.
Present drug development in mitochondrial diseases focuses mainly on modulation of oxidative stress, regulation of the expression of genes involved in metabolic pathways, modulation of coenzymes, induction of mitochondrial biogenesis, and energy replacement.
In this short review, we present the current landscape of mitochondrial disease drug development, focusing on small molecules in clinical trials conducted by industrial sponsor. • Keywords: mitochondrial disease, clinical development, mechanism of action, small molecules, industrial sponsors • Bioblast editor: Tindle-Solomon L • O2k-Network Lab: SE Lund Elmer E
Labels: MiParea: mt-Medicine, Pharmacology;toxicology Pathology: Inherited Stress:Mitochondrial disease Organism: Human