New prospects fot the treatment of Spinal Muscular Atrophy

Poleszak Julita, Szabat Przemysław, Szabat Marta, Boreński Grzegorz, Wójcik Magdalena, Milanowska Joanna, Rejdak Konrad. New prospects fot the treatment of Spinal Muscular Atrophy. Journal of Education, Health and Sport. 2019;9(8):585-596. eISSN 2391-8306. DOI http://dx.doi.org/10.5281/zenodo.3382433

http://ojs.ukw.edu.pl/index.php/johs/article/view/7353

 

 

 

 

 

The journal has had 7 points in Ministry of Science and Higher Education parametric evaluation. Part B item 1223 (26/01/2017).

1223 Journal of Education, Health and Sport eISSN 2391-8306 7

 

© The Authors 2019;

This article is published with open access at Licensee Open Journal Systems of Kazimierz Wielki University in Bydgoszcz, Poland

Open Access. This article is distributed under the terms of the Creative Commons Attribution Noncommercial License which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author (s) and source are credited. This is an open access article licensed under the terms of the Creative Commons Attribution Non commercial license Share alike.

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The authors declare that there is no conflict of interests regarding the publication of this paper.

 

Received: 05.08.2019. Revised: 15.08.2019. Accepted: 31.08.2019.

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

New prospects fot the treatment of Spinal Muscular Atrophy

 

Julita Poleszak^1*, Przemysław Szabat¹, Marta Szabat², Grzegorz Boreński², Magdalena Wójcik², Joanna Milanowska³, Konrad Rejdak⁴

 

¹Students Scientific Society of Neurology, Medical University of Lublin, Poland
²Student Science Club at the Department of Applied Psychology, Medical University of Lublin
³Department of Applied Psychology, Medical University of Lublin
⁴Department of Neurology, Medical University of Lublin, Poland

* E-mail adres: julita.poleszak@wp.pl

 

ORCID ID:

Julita Poleszak https://orcid.org/0000-0002-5166-6262
Przemysław Szabat https://orcid.org/0000-0001-5796-1900
Marta Szabat https://orcid.org/0000-0001-6309-2027
Grzegorz Boreński https://orcid.org/0000-0002-5359-7555
Magdalena Wójcik https://orcid.org/0000-0002-0999-6284
Joanna Milanowska https://orcid.org/0000-0001-9741-1583
Konrad Rejdak https://orcid.org/0000-0002-7019-6681

 

Abstract

Introduction: Spinal muscular atrophy (SMA) is one of the most common genetically determined causes of infant and young child death.

The aim of the study: Review of medical literature on therapeutic strategies used in the treatment of SMA.

Material and method: Standard criteria were used to review the literature data. The search of articles in the PubMed database was carried out using the following keywords:… ..

Description: Therapeutic mechanisms in SMA are directed to: 1) modification of pre-mRNA splicing of the SMN2 gene; 2) gene therapy; 3) neuroprotection; 4) improving skeletal muscle function.

Summary: The gigantic advances in the development of therapeutical strategies resulted in the approval of two drugs for the treatment of SMA and the commencement of clinical studies on numerous with bundles.

 

Keywords: nusinersen, zolgensma, reldesemtiv