Towards a treatment for mitochondrial disease : current compounds in clinical development
(2022) In Bioenergetics Communications 2022(4). p.1-10- Abstract
- 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 cellular 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 clinical signs.
There are currently no cures for mitochondrial diseases, nor any approved pharmaceutical treatments for multisystemic disorders.
Current drug development in mitochondrial diseases... (More) - 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 cellular 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 clinical signs.
There are currently no cures for mitochondrial diseases, nor any approved pharmaceutical treatments for multisystemic disorders.
Current 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 sponsors. (Less)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/c152fe73-8e9a-4a1c-975c-d92f817992be
- author
- Åsander Frostner, Eleonor LU ; Simon Serrano, Sonia LU ; Chamkha, Imen LU ; Elmer, Eskil LU ; Donnelly, Ellen and Hansson, Magnus LU
- organization
- publishing date
- 2022-06-28
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- mitochondria, myopathy, MELAS, primary mitochondrial disease, genetic disorders
- in
- Bioenergetics Communications
- volume
- 2022
- issue
- 4
- pages
- 1 - 10
- ISSN
- 2791-4690
- DOI
- 10.26124/bec:2022-0004
- language
- English
- LU publication?
- yes
- id
- c152fe73-8e9a-4a1c-975c-d92f817992be
- date added to LUP
- 2022-08-05 15:30:00
- date last changed
- 2023-05-15 09:33:28
@article{c152fe73-8e9a-4a1c-975c-d92f817992be, abstract = {{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 cellular 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 clinical signs.<br/><br/>There are currently no cures for mitochondrial diseases, nor any approved pharmaceutical treatments for multisystemic disorders.<br/><br/>Current 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.<br/><br/>In this short review, we present the current landscape of mitochondrial disease drug development, focusing on small molecules in clinical trials conducted by industrial sponsors.}}, author = {{Åsander Frostner, Eleonor and Simon Serrano, Sonia and Chamkha, Imen and Elmer, Eskil and Donnelly, Ellen and Hansson, Magnus}}, issn = {{2791-4690}}, keywords = {{mitochondria; myopathy; MELAS; primary mitochondrial disease; genetic disorders}}, language = {{eng}}, month = {{06}}, number = {{4}}, pages = {{1--10}}, series = {{Bioenergetics Communications}}, title = {{Towards a treatment for mitochondrial disease : current compounds in clinical development}}, url = {{http://dx.doi.org/10.26124/bec:2022-0004}}, doi = {{10.26124/bec:2022-0004}}, volume = {{2022}}, year = {{2022}}, }