期刊
JOURNAL OF CLINICAL MEDICINE
卷 10, 期 1, 页码 -出版社
MDPI
DOI: 10.3390/jcm10010094
关键词
mitochondrial diseases; therapies; mitochondrial toxicity
资金
- Telethon Grant [GUP09004]
- Telethon-MITOCON grant [GSP16001, RF-2016-02361495]
- EJPRD2019 project GENOMIT
MDs are a group of genetic disorders characterized by clinical heterogeneity, treated with supportive therapy due to the lack of specific disease-modifying treatment. Management focuses on early treatment of complications and avoiding harmful drugs.
Mitochondrial diseases (MDs) are a group of genetic disorders that may manifest with vast clinical heterogeneity in childhood or adulthood. These diseases are characterized by dysfunctional mitochondria and oxidative phosphorylation deficiency. Patients are usually treated with supportive and symptomatic therapies due to the absence of a specific disease-modifying therapy. Management of patients with MDs is based on different therapeutical strategies, particularly the early treatment of organ-specific complications and the avoidance of catabolic stressors or toxic medication. In this review, we discuss the therapeutic management of MDs, supported by a revision of the literature, and provide an overview of the drugs that should be either avoided or carefully used both for the specific treatment of MDs and for the management of comorbidities these subjects may manifest. We finally discuss the latest therapies approved for the management of MDs and some ongoing clinical trials.
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