Spinal muscular atrophy – problems of pathogenesis and choice of treatment

Authors

  • Dmitry Kolisnyk Bohomolets National Medical University Tarasa Shevchenka blvd., 13, Kyiv, Ukraine, 01601, Ukraine https://orcid.org/0000-0002-6767-3002
  • Natalia Turchyna Bohomolets National Medical University Tarasa Shevchenka blvd., 13, Kyiv, Ukraine, 01601, Ukraine

DOI:

https://doi.org/10.15587/2519-4798.2017.107795

Keywords:

SMA, SMN1, splicing, axonal transport, valproic acid, antisense oligonucleotides, Nusinersen

Abstract

Aim. The use of inhibitors of histones deacetylase, especially valproates together with the vitamin therapy and the complex of physical exercises was the main method of the treatment of patients with SMA till today. The elaboration of the first, officially acknowledged FDA preparation for SMA treatment forces to review the expedience of the complex therapy using valporoic acid, vitamin additives and physical exercises.

 Methods. The study included 12 patients with different forms of amyotrophy. Depending on the complex of received therapeutic arrangements, they were divided in 4 groups. The treatment effectiveness was estimated in points of Hammersmith scale of the motor function. The comparison of the treatment effectiveness in different groups of patients was realized with the calculation of Student coefficients. 

Result., Motor possibilities of patients in all groups essentially differed from each other depending on SMA type. The number of points, received by patients with the same SMA type, almost didn’t differ depending of a group of patients.  

Conclusions. Motor possibilities of patients are mainly connected with their initial phenotype, only partially influenced by the used treatment methods. The effectiveness of medical means at SMA depends not on the quantity of selected groups of preparations and additional treatment methods but on the initial phenotype

Author Biography

Natalia Turchyna, Bohomolets National Medical University Tarasa Shevchenka blvd., 13, Kyiv, Ukraine, 01601

PhD, Associate Professor

Department of Neurology

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Published

2017-07-31

How to Cite

Kolisnyk, D., & Turchyna, N. (2017). Spinal muscular atrophy – problems of pathogenesis and choice of treatment. ScienceRise: Medical Science, (7 (15), 15–20. https://doi.org/10.15587/2519-4798.2017.107795

Issue

No. 7 (15) (2017)

Section

Medical Science

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Copyright (c) 2017 Dmitry Kolisnyk, Natalia Turchyna

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