Background: Duchenne muscular dystrophy (DMD) has, for the first time, several mutation-specific drugs in clinical use. Four exon-skipping antisense oligonucleotides and one adeno-associated virus (AAV) micro-dystrophin gene therapy carry US FDA approval, and one nonsense-readthrough agent (ataluren) holds a conditional European authorisation that has since been placed under review; ataluren has never been approved by the FDA. Regulatory approval, however, has not translated cleanly into demonstrable clinical benefit, and in resource-limited settings it has not translated into access at all. Methods: We compiled published efficacy estimates for each approved therapy: the EMBARK randomised trial for delandistrogene moxeparvovec (Elevidys), the STRIDE registry for ataluren, and the long-term extension studies of eteplirsen, golodirsen, viltolarsen and casimersen. We read these alongside mutation-spectrum and clinical data from a Kazakh DMD cohort (n = 34), one of the first Central Asian populations to be characterised systematically. Because these sources differ in design, population, endpoint and follow-up, the comparison is indirect and descriptive rather than pooled or statistical. On that basis we assembled three descriptive maps: biological versus clinical efficacy, eligibility versus access, and an order-of-magnitude estimate of cost per year of preserved ambulation. Results: Across the approved therapies, larger biological dystrophin restoration did not correspond to longer preservation of walking. We describe this as the absence of the expected positive association rather than as evidence of a negative one: the comparison rests on a small number of drugs whose values come from heterogeneous studies. We did not—and with these data could not—test it as a formal correlation. Ataluren, with roughly 2% dystrophin restoration, was associated with a longer reported delay in loss of ambulation than Elevidys, which restores 34–51% micro-dystrophin. In the Kazakh cohort, 26.5% of patients were biologically eligible for exon-skipping and 11.8% for ataluren, yet realistic access to any mutation-specific therapy was effectively zero as of April 2026. Our order-of-magnitude cost estimates ran from about $12,000 per additional year of ambulation for standard of care to about $1.3 million for AAV gene therapy, a hundredfold range that did not track clinical effect. Conclusions: We argue that the conventional priority ordering (gene therapy first, exon-skipping second, standard care as background) does not hold up when weighed against patient-relevant outcomes and cost, and may reasonably be inverted for resource-limited systems. This is our interpretation of an indirect comparison, not an evidence-based clinical recommendation. On that reading, the highest-value investments for Central Asia are early molecular diagnosis, universal access to glucocorticoids and specialised physiotherapy, and individual-import pathways for ataluren, while AAV gene therapy is, in our view, a lower near-term priority until its durability and safety data improve.

Background/Objectives: Bronchopulmonary dysplasia (BPD) remains a major complication of prematurity, particularly among infants requiring prolonged respiratory support. Despite its clinical relevance, data on BPD epidemiology and risk factors in Kazakhstan are limited. This study aimed to assess the incidence of BPD and identify significant antenatal, intranatal, and neonatal risk factors associated with its development among preterm infants in Almaty. Methods: A retrospective analysis was conducted using medical records of preterm infants born at the City Perinatal Center in Almaty between 2013 and 2017. Clinical, obstetric, and neonatal data were collected for 111 infants diagnosed with BPD and 109 matched controls without BPD. Statistical analyses included χ² tests, Student’s t-tests, and multiple logistic regression to determine independent predictors of BPD. Results: Among 39,255 live births over a five-year period, 6271 were preterm, and bronchopulmonary dysplasia (BPD) was diagnosed in 111 preterm infants (1.8%). Mortality among BPD infants was 31.9%, with a declining trend over time. Significant maternal factors associated with BPD included edema during pregnancy, polyhydramnios, acute respiratory viral infection (ARVI) during the second trimester, low placentation, vaginal bleeding, chronic bronchitis, and a history of preterm birth. Logistic regression identified edema (p = 0.009) and antenatal steroid prophylaxis (p < 0.001). Neonatal risk factors significantly associated with BPD included neonatal respiratory distress syndrome (p = 0.002), reintubation (p = 0.046), high FiO₂ requirements, prolonged mechanical ventilation, and HFOV use. Regression analysis confirmed neonatal RDS (p = 0.006) and higher FiO₂ (p < 0.001). Conclusions: BPD in Almaty is driven by a combination of maternal complications and severe neonatal respiratory pathology. Early identification and targeted perinatal management of these parameters may help reduce BPD incidence and improve outcomes for preterm infants.

Background: Longitudinal evidence on perinatal and infant mortality in Central Asia remains limited. We described prematurity, the liveborn birthweight distribution, survival of extremely low birthweight (ELBW, 500–999 g) infants, and mortality at a tertiary perinatal center in Turkistan, Kazakhstan. Methods: This single-center ecological study used annual aggregate data for 2016–2025, covering 94,337 births, including 93,482 live births. Poisson models estimated trends; risk ratios (RRs) compared 2025 with 2016. Results: The stillbirth rate was 9.06 per 1000 total births (95% confidence interval [CI] 8.47–9.69) and infant mortality was 4.62 per 1000 live births (95% CI 4.20–5.08). Between 2016 and 2025, perinatal mortality fell from 12.58 to 6.99 per 1000 (RR 0.56, 95% CI 0.41–0.75), stillbirth from 9.29 to 5.42 per 1000 (RR 0.58, 95% CI 0.41–0.82), and infant mortality from 7.66 to 3.57 per 1000 (RR 0.47, 95% CI 0.31–0.70). Preterm births made up 7.1% of births but 66.2% of stillbirths; ELBW infants accounted for 50.2% of infant deaths. Recorded ELBW survival was 21.9% (7/32) in 2016 and 53.8% (28/52) in 2025, but the pooled five-year comparison was not significant (39.9% vs. 47.0%, p = 0.180), and the annual trend lost significance when the 2025 cohort, whose follow-up was incomplete, was excluded. Deaths after discharge were not systematically ascertained. Conclusions: Mortality was lower at the end of the decade, and deaths remained concentrated among preterm and ELBW infants. The ELBW survival findings are hypothesis-generating.