Spinal muscular atrophy: reality and future in Ukraine
Summary. The aim was to clarify peculiarities of spinal muscular atrophy (SMA) natural history in Ukraine and peculiarities, which are caused by medical management. Objective of the study. The research has been done on the analysis of 267 SMA patients’ records, among them were 69 patients of type I, 168 — II and 30 — III. Source of these records was Ukrainian spinal muscular atrophy registry, which is ran by Kharkiv Charitable foundation «Children with spinal muscular atrophy». Results. Such indexes as time to diagnosis, motor functional limitations, the severity of respiratory abnormalities and equipment availability were calculated. Assessments of disease progression speed, lifespan and mortality in different SMA types have been done. Conclusion. The registries are an important source of data for epidemiological and pharmaco-economic researches and a valuable tool for improvement of healthcare. Indexes, mentioned above, despite of positive dynamics, are unacceptable, and their improvement requires organizational and financial investments.
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