A pilot study of idebenone in the treatment of patients with hereditary myopathies.
Summary: The purpose of the study was to evaluate the effectiveness of idebenon in hereditary neuromuscular pathology.
Conclusion: In the general group of patients, there was an increase in muscle strength, although it did not reach statistical significance.
Objective: To evaluate the efficacy of the energotropic drug idebenon in hereditary neuromuscular pathology.
Methods: A total of 9 patients with hereditary myopathies were examined during treatment with idebenone. Determination of muscle strength was carried out on the British medical research Council scale (0 points - no active movements, 5 points - no muscle weakness). The examination was performed before and 1 month after the start of treatment. Treatment regimen: idebenone was prescribed in a daily dose of 90 mg.
Results: In the general group of patients with hereditary ataxia, the median strength of the biceps muscle of the shoulder during treatment increased from 3.5 to 4.0 points (p>0.05). The median strength of the ilio-lumbar muscle increased from 3.0 to 4.0 points (p>0.05). Before treatment, the median number of sit-ups was 0, and after 1 month - 2 (p>0.05).
Conclusions: We used the energotropic drug idebenon, a synthetic analog of the natural substance coenzyme Q10, which is involved in electron transfer in the mitochondrial respiratory chain complex III, for the treatment of patients with hereditary myopathies. In the general group of patients, there was an increase in muscle strength, although it did not reach statistical significance. Before the advent of energotropic drugs, the use of various methods of treating hereditary myopathies did not lead to an increase in muscle strength in patients. Therefore, the identified positive dynamics is of great importance in providing medical care to these patients and improving their quality of life.