Background. Lean production is mainly focused on increasing value in processes and improving the quality of services provided. These principles are implemented in medical organizations within the framework of the national project “Healthcare” and the federal project “Development of the Primary Healthcare System”. Introduction of lean technologies at all levels of the organization can serve as a highly effective method of positive changes. In 2023, Kuban State Medical University, together with the Ministry of Health of Krasnodar Krai and the Regional Center for Primary Healthcare, held a strategic session aimed at identifying problematic processes in medical organizations of the region and jointly searching for ways to solve them. The session involved 73 primary healthcare organizations. Its results formed the basis for a study of the project activity of medical organizations. Objective. To analyze the current state of project activities of medical organizations in Krasnodar Krai to achieve a “new model” through the introduction of lean technologies.
Methods. A one-stage observational study was carried out in two steps. The first step involved participant observation. Its results determined a set of tools to be compiled. The second step of the study included a questionnaire survey and analysis of information. The data obtained from the questionnaire survey and observation were coded, the distribution and frequency were identified using frequency analysis and calculation of mean values. The qualitative analysis of open-ended questions was additionally applied.
Results. The analysis showed that the prevailing number of projects are aimed at forming and developing the availability of medical care for the population (50.6 %), including projects focused on inclusiveness and provision of medical care to low-mobility patients at home. It was also revealed that 42 projects in four areas are planned for implementation in 47 organizations using lean technology tools in 2024. The study showed that 91.49 % of projects failed to calculate financial efficiency and evaluate project results in monetary terms. Meanwhile, 8.51 % of those who carried out the efficiency calculations lacked an analysis methodology and revealed the superficiality of the calculations. Of all the participants, 53.19 % reported the need for help in organizing and implementing project activities, and 36.17 % of the participants had enough resources to cope on their own. The three most problematic processes requiring joint work with colleagues included “Reducing the wait times for healthcare in regional medical organizations,” “Registration of discounted prescriptions” and “Preventive medical examination and dispensary.”
Conclusion. The experience of a strategic session at Kuban State Medical University proved to be effective in improving the quality of implementation of lean technologies in medical organizations of Krasnodar Krai. The study was instrumental in assessing the current state of the project activities of medical organizations to achieve a “new model” through the introduction of lean technologies, as well as in identifying the most common areas of project activities and the main problems. The performed analysis revealed a positive trend in improving the quality of work of medical organizations in Krasnodar Krai and disseminating the lean technologies in primary healthcare.

Background. Infections associated with socially significant diseases considerably contribute to a decrease in the quality of public health. Tuberculosis, though declining in Russia, is still recognized as a pressing public health problem. Meanwhile, an assessment of the burden of tuberculosis on society, using such comprehensive methods as measuring disability-adjusted life years, is yet to be investigated in Russia. Objective. To estimate the tuberculosis burden by calculating the index of disability adjusted life years in a high-density region.
Methods. The retrospective cohort study involved data on newly-diagnosed and died of tuberculosis cases that corresponded to the annual reporting form of federal statistical surveillance No. 8 “Information on active tuberculosis cases” for the Republic of North Ossetia-Alania; information on patients who died of tuberculosis or tuberculosis combined with infection caused by human immunodeficiency virus, was obtained from the Department of Civil Status Records of the Republic of North Ossetia-Alania and verified with data from the Federal State Statistics Service; data on tuberculosis-associated disability was received from Main Bureau of Medical and Social Expertise in the Republic of North Ossetia-Alania of the Ministry of Labor and Social Protection of the Russian Federation for 2018 and 2022. Following the analytic dataset, 691 people with newly-diagnosed tuberculosis, who received TB-associated disability and died from TB in 2018 (318 people) and 2022 (373 people) were identified. The study involved calculation of gender and age standardized TB morbidity and mortality rates as well as DALY index with its components: Years of Life Lost due to tuberculosis and Years Lost due to Disability. The study involved comparing data between 2018 and 2022 and gender/age-specific indicators within the same year. Mathematical data processing was performed using Microsoft Excel 2017 (Microsoft Corporation, USA) and SPSS 26.0 (SPSS Inc., USA). Differences between the compared indicators were evaluated by Chi-square criterion with variations, the initial data were grouped in a contingency table, the significance of differences was expressed by p-value.
Results. The burden of tuberculosis in the Republic of North Ossetia-Alania refers predominantly to the male population: with a gender difference in the decrease in the disability-adjusted life years in 2018 and 2022 by 4.4 and 9.0 times (p < 0.05), respectively; 2.8 times among males, 5.5 times among females (p < 0.05). The burden of tuberculosis is mainly associated with premature mortality due to tuberculosis among males aged 35–54 years and among females aged 35–44 years with a pronounced decrease in Years of Life Lost. Years of Life Lost among males aged 20–34 in 2022 appears two times higher than the cohort average. The increase in Years of Life Lost among males aged 60–64 accounted for 1.7 times, in Years Lost Due to Disability — 11.2 times (p < 0.05). Years Lost Due to Disability among females aged 20–44 years appeared to be the highest, exceeding the cohort average by more than two times in 2018.
Conclusion. The burden of tuberculosis is predominantly due to tuberculosis-related premature mortality among the male population. According to the disturbing conclusion, Years of Life Lost among young males aged 20–34 years and tuberculosis-related premature mortality shift towards the male population of pre-retirement age (60–64 years) with an increase in Years Lost Due to Disability among them. Notably, the study reveals high rates of Years Lost Due to Disability among females aged 20–44. The results obtained should be taken into account when developing regional tuberculosis programs with their targeted use in “vulnerable” age and gender groups of the population.

Background. Postpartum hemorrhage is recognized as a leading cause of maternal mortality and morbidity in the world. Predicting postpartum hemorrhage in high-risk patients with uterine scar enables preventive measures to be scheduled and costs of treatment and rehabilitation to be reduced.
Objective. To determine antenatal predictors of high postpartum hemorrhage risk in pregnant women with uterine scar in order to improve the prevention of massive blood loss.
Methods. An observational cohort study involves the medical records of 4494 maternity women with uterine scar (pregnancy and delivery histories) of the Perinatal Center of Regional Clinical Hospital No. 2, Krasnodar Krai. The study sample included data of maternity women coded O34.2 according to International Classification of Diseases, 10th Edition, for the period from 2017 to 2020. The sample participants were distributed into two groups depending on the blood loss during delivery, determined in compliance with clinical recommendations: a group of patients without massive blood loss during delivery and a group of patients with massive blood loss in labor/early postpartum period. The study was mainly focused on parameters of the prediction performance of bleeding in labor and early postpartum period in pregnant women with uterine scar using a multiparametric, logistic regression models. The study considered demographic data, comorbidity, obstetric history, pregnancy course, ultrasound data, and volume of blood loss at delivery. The performance of prediction for postpartum hemorrhage was calculated using multivariate binary logistic regression. Descriptive statistical analysis was carried out by means of statistical software package SPSS Version 26 (IBM, USA). Two-sided p-value < 0.05 was taken as a statistically significant difference. A prognostic significance of predictors was determined by binary logistic regression. The Wald statistic was used to determine an observed significance. In order to define the performance of the model, the study involved calculating sensitivity, specificity, positive and negative predictive value, Nagelkerke coefficient of determination, as well as performing ROC analysis. The DeLong test was used for paired comparisons of ROC curves.
Results. In the retrospective follow-up group (2017–2020) (n = 502), postpartum hemorrhage with massive blood loss occurred in 41 cases (8.17%). For the model based on clinical-anamnestic predictors, the prediction performance for postpartum hemorrhage comprised: sensitivity = 12.2% (95% confidence interval (CI) 4.1–26.2); specificity = 99.3% (95% CI 98.1–99.9); positive predictive value = 62.5% (95% CI 24.5–91.5); negative predictive value = 92.6% (95% CI 89.9–94.8); area under the ROC curve = 0.864 (95% CI 0.807–0.920), p < 0.001. The prediction performance for the model based on three ultrasound predictors (asymmetry coefficient of placental thickness, uterine wall bulging in the scar and myometrial thickness in the placentation zone) comprised: sensitivity = 85.4% (95% CI 70.8–94.4); specificity = 98.5% (95% CI 96.9–99.4); positive predictive value = 83.3% (95% CI 68.6–93.0); negative predictive value = 98.7% (95% CI 97.2–99.5); area under the ROC curve = 0.919 (95% CI 0.855–0.983), p < 0.001. No significant difference was revealed for the performance of the models ( p = 0.170). For the model based on ultrasound predictors and placenta previa, the prediction performance comprised: sensitivity = 85.4% (95% CI 70.8–94.4); specificity = 98.5% (95% CI 96.9–99.4); positive predictive value = 83.3% (95% CI 68.6–93.0); negative predictive value = 98.7% (95% CI 97.2–99.5); area under the ROC curve = 0.955 (95% CI 0.912–0.999), p < 0.001. The model based on clinical-anamnestic and ultrasound indicators predicted postpartum hemorrhage with sensitivity equal to 85.4% (95% CI 70.8–94.4); specificity — 98.9% (95% CI 97.4–99.6); positive predictive value — 87.5% (95% CI 73.2–95.8); negative predictive value — 98.7% (95% CI 97.1–99.5); area under the ROC curve — 0.984 (95% CI 0.966–1.0), р < 0,001. Thus, this model outperformed the model based on clinical-anamnestic data (p < 0.001), based on ultrasound predictors (p = 0.006) and revealed no difference with the model considering placenta previa and ultrasound predictors (p = 0.127). Using prenatal prediction of postpartum hemorrhage based on ultrasound features, the incidence of massive blood loss at delivery decreased from 6.88/1000 deliveries (2019–2020) to 4.18/1000 deliveries (2021–2022) (p < 0.001).
Conclusion. Ultrasound predictors in pregnant women with uterine scar increase the sensitivity of antenatal prediction of postpartum hemorrhage based on the assessment of clinical and anamnestic risk factors, thereby enabling preventive measures to be scheduled in the risk group and incidence of massive blood loss to be reduced.

Background. A microbial nature of periodontitis requires an accurate analysis of the pathogenic potential of the periodontal pocket microflora thereby enabling correct diagnosis to be made and the most effective pharmacological agent to be selected for treating chronic periodontitis. Metronidazole demonstrates excellent results for anaerobic microorganisms in the periodontal pocket, whereas minocycline hydrochloride, moxifloxacin and amoxicillin/clavulanate are efficient for aerobic microorganisms. However, specialists find considerably relevant to analyze the correlations between specific bacterial associations in the periodontal pocket against local antibacterial therapy, as well as to track the contents of periodontal pockets long after antibacterial therapy. Therefore, a microbiological analysis of periodontal pocket contents is considered necessary in order to select and monitor the effectiveness of local antibacterial therapy.
Objective. To evaluate the effectiveness of local antibacterial therapy for chronic generalized periodontitis by means of analysis of changes in the microbiological composition of periodontal pockets.
Methods. The clinical trial involved 144 patients with chronic generalized periodontitis. The study included examination of patients and analysis of clinical data bases of the Department for General Practice Dentistry, Kuban State Medical University and the Dentistry Department, Stavropol State Medical University of the Ministry of Health of the Russian Federation. The trial included patients aged 30 to 60 with a diagnosis of chronic generalized periodontitis of different severity. All periodontitis patients were divided into 2 groups depending on the therapy: main and control groups. Each group had three subgroups depending on the severity of the pathologic process. The first subgroup of the control group (chronic generalized periodontitis of mild degree) consisted of 16 people (11.1%), the second subgroup (chronic generalized periodontitis of moderate degree) — 31 people (21.5%), the third subgroup (chronic generalized periodontitis of severe degree) — 7 people (4.9%). The first, second and third subgroups of the main group consisted of 28 (19.4%), 48 (33.3%) and 14 persons (9.7%) respectively. The severity of periodontitis was determined by the depth of the periodontal pocket, degree of bone resorption (according to x-ray examination), and pathologic tooth mobility. A local anti-inflammatory therapy for the patients of the control group included oral baths with propolis (15–20 drops of 10% propolis tincture diluted in 100 ml of warm water) 3 times a day for 7 days starting from the first visit. At the final stage of treatment after preliminary scaling and rootplaning, patients of the control group received antiseptic gel with 10% metronidazole and 2% chlorhexidine bigluconate to be injected into periodontal pockets with over 4 mm depth. Additionally, applications of adhesive dental films Diplene Denta L (with lincomycin) and Diplene Denta M (with metronidazole) were prescribed to be done in the home. In addition to the above-mentioned comprehensive therapy, patients of the main group received a single insufflation of minocycline hydrochloride microspheres into each periodontal pocket. The tools for the patients of the main group included plastic cannulas with minocycline hydrochloride microspheres in the form of powder in the tip of the cartridge, and a special syringe-insufflator. The technique of antibiotic insufflation into the periodontal pocket was preliminary trained on the model of jaws with the studied pathology. Statistical analysis of the results was performed using variance analysis in the Excel Microsoft Office 2016 (Microsoft, USA).
Results. According to the systematic observation of the patients from the control group, periodic fluctuations in microbial population in the majority of pathologic pockets obtained a specific regularity: a decrease in the number of microorganisms on day 21 was followed by its increase in the subsequent period of observation after 30 and especially 60 days. According to the ongoing follow-up of the patients of the main group, fluctuations of microbial population in the majority of pathologic pockets obtained a certain regularity: a significant reduction in the number of microorganisms on day 21 was followed by its slight rise in the subsequent period of observation after 30 and a slight increase again in 60 days. Fluctuations in the number of microorganisms of periodontal pockets in the main group reveal their specific character: their number remained invariably lower than that of the control group throughout the observation period, while the total number of detected periodontopathogens decreased.
Conclusion. The established correlations between the main indicators of microbiological status of periodontal pockets and the severity of chronic generalized periodontitis within different periods after the local therapy confirm the positive dynamics of treatment in the main group, where patients were insufflated with minocycline hydrochloride microspheres, proved by a change in the typology of microbial status of periodontal pockets and a number of periodontopathogenic microorganisms.

Background. According to the hypothesis of the study, the therapeutic efficacy of measures to treat hypoxic (ischemic) emergencies can be significantly increased by using an artificial gas mixture with normal or increased oxygen content and a high content of argon, being bioactive despite its chemical inertness.
Objective. To experimentally evaluate the antihypoxic effects of argon on the example of acute massive blood loss in experimental animals.
Methods. The study was carried out on 72 albino male gray rats with a baseline body weight of 220–250 g. The experiments were performed at the premises of the Konstantinov St. Petersburg Nuclear Physics Institute of the Kurchatov Institute and the Smorodintsev Research Institute of Influenza (Russia). Prior to the experiment, the animals were quarantined for 2 weeks in the vivariums of the performing organizations. Acute massive blood loss in rats was formed by taking blood (12 ± 1% of body weight on average) using syringe method by transcutaneous cardiac puncture. Rats were randomized into 3 groups (24 individuals each) depending on the gaseous medium where the animal was placed after artificial blood loss. Composition of the applied gaseous medium in the comparison groups: medium No. 1 — oxygen 21% vol.; argon — 30% vol.; nitrogen — the rest; medium No. 2 — oxygen 21% vol.; argon — the rest; medium No. 3 (control) — air. The postoperative follow-up (post-conditioning) for 8 hours involved assessing and recording the following indicators: survival/lethality rates; recovery rates (anesthesia recovery, somatic mobilization); clinical symptoms of possible intoxication with the gas mixture during inhalation exposure. The surviving animals were then monitored for 4 days to evaluate their health and behavioral performance. Upon completing the observation period, the animals were euthanized.
Results. After artificially-induced blood loss, all the non-survivors died within 1.5–8 hours after the operation. In group 1 (post-conditioning in medium No. 1), the lethality of laboratory animals amounted to 4 individuals out of 24 (16%); in group 2 (post-conditioning in medium No. 2) — 2 individuals out of 24 (8%). In the control group, the lethality rate was significantly higher — 10 animals out of 24 (42%). The surviving animals demonstrated no lethality or morbidity within 4 days after the exposure. Analysis of outcomes between experimental groups and controls revealed that the significance of differences in the lethality index between group 1 and control (by Fisher’s two-sided criterion) comprised p = 0.06; between group 2 and control — p = 0.017. When the statistical “power” of the experimental group was increased (by combining samples 1 and 2), the significance of differences in outcomes in this group compared to the control accounted for p < 0.001.
Conclusion. The conducted studies confirmed the hypothesis about the pronounced antihypoxic effect of argon, which significantly increased the survival rate of experimental animals after massive artificially-induced blood loss.

Background. Stressful influences, depending on their severity and duration, can cause the development of pathological conditions. Repeated episodes of stress cause functional and structural changes in the central nervous system and can cause the development of depressive conditions. Depression is one of the leading mental illnesses. One of the most stress-sensitive brain structures is the hippocampus. Objective. To study is to evaluate structural changes in the hippocampus, which is considered as a heterogeneous structure with separate dorsal and ventral regions, to evaluate the expression of inducible nitric oxide synthase, endothelial nitric oxide synthase, serine racemase, synaptophysin in a mild stress model.
Methods. A study of the effects of mild stress was carried out on 16 adult male Wistar rats (age 12 months, body weight 350–400 g). After acclimatization, the rats were divided into two equal groups (n = 8): intact (control) and stressed. When keeping animals, modeling and removing them from the experiment, we were guided by the Regulations for Carrying Out Work Using Laboratory Animals and the Declaration of Helsinki. Experimental modeling of depression in animals was induced by mild stress exposure for 7 days (30 minutes daily). Euthanasia was performed in a CO2 incubator. The brain was fixed in neutral buffered 10% formalin. Paraffin sections were made in the frontal plane, stained with hematoxylin and eosin, thionin using the Nissl method and examined at a level from –2.40 to –3.96 mm relative to bregma using an Axio Lab A1 microscope (Carl Zeiss Microscopy GmbH, Germany). Photo documentation was carried out with an AxioCam 105 color camera (Carl Zeiss Microscopy GmbH, Germany). Using the Image Analysis module of the ZEN 1.1.2.0 program (Carl Zeiss Microscopy GmbH, Germany) in the pyramidal layer of the hippocampus. Statistical analysis was performed with Microsoft Office Excel 2016 (Microsoft, USA) and Prism 6 (GraphPad Software Inc., USA). Comparisons of two conditions were made by nonparametric Mann-Whitney-U test to avoid a statistical bias of unequal data distribution. The level of significance was set at p < 0.05. The summarized data were presented as a as mean ± standard error of mean.
Results. Functional research methods and assessment of pathological changes in hippocampal neurons are presented. An increase in the relative number of wrinkled hyperchromatic pyramidal neurons in the dorsal cornu ammonis field 3 in stressed rats was noted by 23.6% (p < 0.05) compared to the control. There was an increase in the relative number of inducible nitric oxide synthase-immunopositive neurons in the dorsal cornu ammonis field 3 by 40% ( p < 0.05) and the relative area of inducible nitric oxide synthase-immunoreactive material by 35% (p < 0.05) in the pyramidal layer of cornu ammonis field 3 in stressed rats. A decrease in the relative area of synaptophysin-immunopositive material in stressed rats was found in the ventral cornu ammonis field 3 compared to the control group by 16.8% (p < 0.05); decrease in the relative area of serine racemase-immunopositive material in dorsal cornu ammonis field 3 by 4.3% (p < 0.05) and ventral cornu ammonis field 3 by 7.8% (p < 0.05).
Conclusion. The results of the study demonstrate that mild stress is an adequate model of depression in rats. In animals exposed to mild stress, pronounced morphological signs of damage to hippocampal neurons were revealed; motor and indicative exploratory activity decreases. Differences were found in morphofunctional changes in the dorsal and ventral parts of the hippocampus under the influence of mild stress. In cornu ammonis field 3 of the dorsal hippocampus, in contrast to the ventral section, more pronounced signs of damage to pyramidal layer neurons were observed. The increase in the relative number of inducible nitric oxide synthase-immunopositive neurons and the relative area of inducible nitric oxide synthase-immunoreactive material in the cornu ammonis field 3 pyramidal layer in stressed rats indicates an increase in nitric oxide production and the participation of nitrooxide-dependent free radical mechanisms of damage to hippocampal neurons. The decrease in the relative area of synaptophysin-immunoreactive material in stressed rats may contribute to changes in synaptic plasticity. A decrease in the relative area of serine racemase-immunoreactive material in the dorsal and ventral parts of cornu ammonis field 3 is considered to be a sign of a possible decrease in N-methyl-D-aspartate-dependent neurotransmission in the hippocampus under stress.

Background. Autophagy refers to an evolutionary physiological mechanism of cell self-purification. The use of its positive effects in regenerative and preventive medicine implies this process to be investigated under the conditions of various therapeutic factors.
Objective. To investigate the influence of gender, age and somatic-morphological factors on the autophagy processes induced by a comprehensive wellness program as expressed by the marker protein Beclin-1.
Methods. A prospective observational study involved 107 patients to be monitored before and after comprehensive wellness procedures. All study participants underwent a wellness program in the clinic of OOO Centr Zdorovie (Maykop, Russia). The study was conducted in the periods of September‒December 2020 and January 2021. The study involved measuring the concentration of Beclin-1 protein in the blood before and 12 days after the wellness procedures. In order to characterize the degree of activation of the autophagy process, the index “delta-Beclin-1” was introduced (difference between the initial level of Beclin-1 protein concentration and the level registered after 12 days). The autophagy response was evaluated in terms of its upward or downward direction (positive/negative delta- Beclin-1 value, respectively). The effect of the comprehensive wellness procedures on the functional systems of the organism was determined in accordance with the laboratory tests (complete blood count, lipid profile), morphometric evaluation, and the assessment of body composition by impedance monitoring. The obtained data were processed using SPSS Statistics 26.0 (IBM, USA).
Results. Monitoring of Beclin-1 protein concentration revealed that basic autophagy activity is age-specific and significantly lower in individuals over 60 years as compared to both individuals under 60 years and healthy persons. This correlation with age appears more pronounced in obese individuals (r = -0.59). The basic activity of autophagy in men was 30% lower than in women, however, after a set of wellness procedures the mentioned response in men increased by 7%. The study found that not all patients responded unidirectionally to the comprehensive wellness procedures: 49 out of 77 patients showed an increase in Beclin-1 protein concentration, while 28 — a decrease. Patients who participated in the wellness program demonstrated negative correlation between the level of Beclin-1 protein expression and age, while those responding with an increase in Beclin-1 protein level — with body mass index, weight and fat mass. The source of autophagy activity in men under 60 years can refer to fat mass, and in women — lean body mass, especially in the mature age of period II (36‒60 years).
Conclusion. The factors of basic autophagy and autophagy induced by the complex of wellness procedures include age, gender and body weight. These factors obtain different significance at different age periods. A comprehensive wellness program can serve as an alternative to existing pharmacological methods for activating autophagy in humans.

Background. Infantile-onset form of Pompe disease (IOPD) comprises a progressive and fatal disease in the absence of pathogenetic treatment. Enzyme replacement therapy using biologically active recombinant human alglucosidase alfa is considered as a treatment option to increase the life expectancy of patients with early diagnosis and timely initiation of therapy.
Case description. A child aged 5 months was delivered to the Cardiology Department of Children’s Republican Clinical Hospital (Tatarstan, Russia) for examination and clarification of the diagnosis. The patient’s parents complained of his shortness of breath, unproductive cough, cyanosis of the nasolabial triangle when restless, weakness, lethargy during breastfeeding session, as well as decreased appetite up to refusal to eat, constantly enlarged tongue. The examination revealed macroglossia and hypersalivation, pseudohypertrophy of calf muscles, dyspnea involving the accessory muscles, hepatomegaly, diffuse muscular hypotonia. Laboratory tests revealed an increase in alanine aminotransferase level greater than four times the normal values, aspartate aminotransferase level — more than nine times, a significant rise of creatine phosphokinase and lactate dehydrogenase, as well as natriuretic peptide. Echocardiography revealed significant myocardial hypertrophy of both the left and right ventricles. Complaints, medical history, clinical examination, and all of the above changes in laboratory and instrumental examinations suggested a group of hereditary metabolic diseases (Pompe disease), confirmed by a pronounced decrease in the activity of the alpha-glucosidase enzyme to 0.12 μmol/l/h in dry blood spots with subsequent molecular genetic study and detection of compound-heterozygous mutations in the GAA gene. It was decided to initiate specific enzyme replacement therapy by intravenous administration of a recombinant form of human acidic alpha-glucosidase (alglucosidase alfa) at a dose of 20 mg/kg/injection in a frequency of once every 2 weeks. In addition, CRIM (cross-reactive immunological material)-negative status determined immune tolerance therapy, including the most studied combination of three drugs: rituximab, methotrexate, and intravenous immunoglobulin, as a response to enzyme replacement therapy. Long-term observation of the effect of enzyme replacement therapy brought positive results in the form of new motor skills, decreased content of intracellular enzymes and natriuretic peptide, reduction of myocardial hypertrophy of the left and right ventricles. In the future, the child needs lifelong treatment.
Conclusion. Awareness of physicians about Pompe disease will prevent the growth in diagnostic errors and neglected cases. In case of early confirmation, the effectiveness of currently available ERT increases: the possibility to stop the disease progression, to reverse its individual clinical manifestations, and to improve the patient’s quality of life.