Back to Search Start Over

Clinical trials in mitochondrial disorders, an update.

Authors :
Almannai, Mohammed
El-Hattab, Ayman W.
Ali, May
Soler-Alfonso, Claudia
Scaglia, Fernando
Source :
Molecular Genetics & Metabolism. Sep2020, Vol. 131 Issue 1/2, p1-13. 13p.
Publication Year :
2020

Abstract

Mitochondrial disorders comprise a molecular and clinically diverse group of diseases that are associated with mitochondrial dysfunction leading to multi-organ disease. With recent advances in molecular technologies, the understanding of the pathomechanisms of a growing list of mitochondrial disorders has been greatly expanded. However, the therapeutic approaches for mitochondrial disorders have lagged behind with treatment options limited mainly to symptom specific therapies and supportive measures. There is an increasing number of clinical trials in mitochondrial disorders aiming for more specific and effective therapies. This review will cover different treatment modalities currently used in mitochondrial disorders, focusing on recent and ongoing clinical trials. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
10967192
Volume :
131
Issue :
1/2
Database :
Academic Search Index
Journal :
Molecular Genetics & Metabolism
Publication Type :
Academic Journal
Accession number :
147604325
Full Text :
https://doi.org/10.1016/j.ymgme.2020.10.002