[Experience of the treatment of spinal muscular atrophy type 3 Kugelberg-Welander with Nusinersen]. / Opyt lecheniya nusinersenom spinal'noi myshechnoi atrofii 3-go tipa Kugel'berga­Velander.

Before the advent of pathogenetic therapy, the diagnosis of spinal muscular atrophy (SMA) meant the loss of all hopes for recovery and the patient's setting on the path of a steady decline in motor functions, a deterioration in the quality of life and, ultimately, inevitable early death. Currently, new methods of pathogenetic therapy with nusinersen and risdiplam, as well as etiological therapy with onasemnogene abeparvovec, are available in the Russia. Nusinersen is an antisense oligonucleotide that modifies splicing of the SMN2 gene to increase production of normal full-length motor neuron survival protein, which is deficient in SMA. The mechanism of action of Nusinersen is based on the activation of the disabled exon 7 of the SMN2 gene. The article describes an example of long-term effective treatment using pathogenetic therapy of a patient diagnosed with SMA type 3.

[The role of inflammation in the development of diabetic polyneuropathy and the possibility of its correction]. / Rol' vospaleniya v razvitii diabeticheskoi polineiropatii i vozmozhnost' ego korrektsii.

OBJECTIVE: To study the effect of the combined drug Cytoflavin on the mechanisms of nonspecific inflammation in the treatment of diabetic polyneuropathy (DPN) with an assessment of the dynamics of the TNF-α index. MATERIAL AND METHODS: An open comparative prospective observation of patients with a history of DPN for more than 5 years and a high level of TNF-α was carried out. All patients underwent basic oral combined hypoglycemic therapy, the main group used the combined drug Cytoflavin 10 ml (per 200 ml 0.9% NaCl) for 10 days, followed by the transition to the enteral form of the drug, 2 tablets 2 times a day for 1 months The main indication for the appointment of Cytoflavin was the presence of comorbid pathology in the form of cerebrovascular disease in all studied patients. The severity of clinical symptoms of DPN, the quality of life (QOL) of patients, as well as the dynamics of the level of TNF-α as an indicator reflecting the process of inflammation were assessed. RESULTS: As a result of the treatment in the study group, there was an improvement in QoL, a decrease in the severity of sensory complaints and a decrease in the level of TNF-α, which may indicate a possible anti-inflammatory mechanism of the combined drug Cytoflavin. CONCLUSION: Cytoflavin can inhibit inflammation and reduce the severity of sensitive disorders in patients with DPN.

[The efficacy and safety study of Mexidol and Mexidol FORTE 250 in patients with chronic cerebral ischemia]. / Effektivnost' i bezopasnost' terapii lekarstvennymi preparatami Meksidol i Meksidol FORTE 250 u patsientov s khronicheskoi ishemiei golovnogo mozga.

OBJECTIVE: To study the efficacy and safety of sequential MexidoL therapy, administered intravenously (500 mg once a day) for 14 days, followed by taking the oral form Mexidol FORTE 250 at a dose of 250 mg (1 tablet) 3 times a day for 60 days in elderly patients with chronic cerebral ischemia (CCI) on the background of arterial hypertension and atherosclerosis. MATERIAL AND METHODS: An open prospective observational study included 60 patients with an established diagnosis (CCI), confirmed by the results of neuroimaging. All patients were examined with an assessment of neuropsychological status (MoCA test), severity of asthenia (scale MFI-20), emotional state (Hamilton anxiety and depression scale), motor functions (formalized clinical scale for assessing motor activity of elderly Tinetti). The effectiveness of the therapy was evaluated according to the quality of life questionnaire (SF-36). RESULTS: The results of the study showed the high efficiency and safety of sequential therapy with Mexidol in relation to the relief of asthenic and emotional disorders, improving the state of cognitive functions, improving the quality of life of patients. The maximum effect occurred after the end of the full course of therapy. High adherence of patients to the therapy, low frequency of adverse events are shown. CONCLUSION: Sequential use of intravenous administration of Mexidol followed by oral administration of Mexidol FORTE 250 is an effective and safe way to treat patients with CCI.

[Results of an international multicenter, randomized, double-blind, placebo-controlled study assessing the efficacy and safety of sequential therapy with Mexidol and Mexidol FORTE 250 in patients with chronic brain ischemia (MEMO)]. / Rezul'taty mezhdunarodnogo mnogotsentrovogo randomizirovannogo dvoinogo slepogo platsebo-kontroliruemogo issledovaniya otsenki effektivnosti i bezopasnosti posledovatel'noi terapii patsientov s khronicheskoi ishemiei mozga preparatami Meksidol i Meksidol FORTE 250 (issledovanie MEMO).

OBJECTIVE: To assess the efficacy and safety of sequential therapy with Mexidol solution for intravenous and intramuscular administration, 50 mg/ml and Mexidol FORTE 250 film-coated tablets, 250 mg in patients with chronic brain ischemia (CBI). MATERIAL AND METHODS: An international multicenter, randomized, double-blind, placebo-controlled trial, conducted in 15 clinical centers located in Russian Federation and Republic of Uzbekistan, included 318 patients with CBI aged 40 to 90 years. The patients were randomized into 2 groups, the patients of the 1-st group received Mexidol intravenously 500 mg once daily for 14 days, followed by Mexidol FORTE 250 - 250 mg 3 times a day orally for 60 days; patients of the 2-nd group received a placebo in a similar mode. The primary endpoint was the mean value of difference by MoCA scale at the point of completing the therapy comparing to initial value. RESULTS: According to the results of the assessment of the primary endpoint, statistically significant changes in the MoCA scores at the stage of completion of study were revealed when comparing the dynamics between the 1-st and 2-nd groups (p<0.000001). The lower limit of the 95% confidence interval for the difference in the average of the main efficacy endpoint between the 1-st and 2-nd groups was 1.51, which allows to state a higher efficacy of the use of Mexidol. According to the estimates of secondary endpoints, a statistically significant advantage over placebo at the last visit achieved while evaluation by the following scales and tests: digit symbol substitution test, MFI-20 asthenia assessment scale, Beck anxiety scale, Vane questionnaire, Tinetti scale, SF-36 questionnaire (mental component of health), CGI scale. The comparable nature of the safety profile of Mexidol and Placebo was established. CONCLUSION: The validity and expediency of the use of Mexidol and Mexidol FORTE 250 in the treatment of patients with CBI has been demonstrated.

[Painful diabetic polyneuropathy: focus on life quality improvement of patients]. / Bolevaia forma diabeticheskoi polineiropatii: fokus na uluchshenie kachestva zhizni patsienta.

AIM: To evaluate the efficacy and safety of cytoflavin as an additional agent in the treatment of painful diabetic polyneuropathy and to analyze changes in the life quality of the patients studied. MATERIAL AND METHODS: An analysis of treatment data was carried out in 61 patients with verified painful diabetic polyneuropathy, who were divided into 2 groups depending on the therapy regimen. Patients of the main group (n=36) at the start of the therapy with gabapentin additionally received cytoflavin: intravenously, slowly, 10 ml diluted in 0.9% NaCl 200 ml for 10 days, followed by switching to per os 2 tablets 2 times a day for 25 days. Patients of the comparison group used gabapentin in comparable doses as an analgesic symptomatic therapy. Clinical neurological and anamnestic methods were used to monitor and assess the condition of patients. RESULTS AND CONCLUSION: Cytoflavin inclusion in the standard symptomatic treatment of patients with painful diabetic polyneuropathy contributed to a more pronounced decrease in the subjective assessment of pain (VAS scale) by the 10th day (42.8±2.4 mm versus 58.2±2.1 mm in the comparison group) and its maximum level of decline to 21-25 days. The achieved result was maintained by the 35th day (21.4±1.1 mm against 22.4±1.7 mm in the comparison group). At the same time, the quality of life of patients as assessed by SF-36 was significantly increased after treatment. The results obtained, along with the safety of the drug, allow us to recommend its inclusion in the treatment regimens for patients with this pathology.

[Optimization of hypolipidemic therapy in patients with ischemic stroke and diabetes mellitus].

OBJECTIVES: Evaluation of effectiveness of Mexidol in optimization of hypolipidemic therapy in ischemic stroke and diabetes mellitus patients. MATERIAL AND METHODS: Authors analyzed the indicators of lipid status: total cholesterol, low-density lipoproteins, high density lipoproteins, triglycerides and concentration of platelet factor-4, ß-tromboglobulin, von Willebrand factor in 68 patients with acute ischemic stroke and diabetes mellitus. Authors investigate the dynamics of these parameters (1(st), 21(st), 3-d and 6(th) month after onset stroke) depending on timing and dose of Mexidol. RESULTS: Long time therapy of Mexidol may optimize of hypolipidemic therapy in ischemic stroke and diabetes mellitus patients.

[Endothelial dysfunction in ischemic strokes in diabetes mellitus].

The progression of an ischemic stroke concerns the activation of endothelial cells especially in patients with diabetes mellitus type 2. The aim of our study was to evaluate the endothelial dysfunction in patients with acute ischemic stroke with diabetes mellitus type 2. We measured the plasma activities of von Willebrand factor (vWf) as endothelial markers. The plasma vWf activities were significantly elevated in the index group compared to patients with ischemic stroke and arterial hypertension and controls. The correlation of the neurologic deficit severity to day 21 with the activity of endothelial dysfunction was revealed as well.