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Updated: Aug 9, 2025
06:05
An In Vitro Approach to Study Mitochondrial Dysfunction: A Cybrid Model
Published on: March 9, 2022
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Clinical trials in mitochondrial diseases.
Amel Karaa1, Thomas Klopstock2
1Mitochondrial Disease Program, Division of Medical Genetics and Metabolism, Massachusetts General Hospital, Boston, MA, United States; Department of Pediatrics, Harvard Medical School, Boston, MA, United States.
Handbook of Clinical Neurology
|February 22, 2023
Summary
Primary mitochondrial diseases, complex inherited metabolic disorders, face slow therapeutic development due to diverse challenges. This review examines current and future drug development strategies for these rare conditions.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Primary mitochondrial diseases are common, complex inherited metabolic disorders.
- Significant challenges including lack of data and biomarkers hinder drug development.
- Recent interest and incentives are driving new therapeutic efforts.
Conclusions:
- Despite challenges, there is growing momentum in developing therapies for primary mitochondrial diseases.
- Future strategies will likely involve addressing data gaps and improving trial design.
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