Clinical trials in mitochondrial disorders, an update.
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ABSTRACT:
SUBMITTER: Almannai M
PROVIDER: S-EPMC7537630 | biostudies-literature | 2020 Oct
REPOSITORIES: biostudies-literature
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Clinical trials in mitochondrial disorders, an update.
Almannai Mohammed M El-Hattab Ayman W AW Ali May M Soler-Alfonso Claudia C Scaglia Fernando F
Molecular genetics and metabolism 20200901 1-2
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 meas ...[more]
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