New prospects for the treatment of Spinal Muscular Atrophy
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...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Kazimierz Wielki University
2019-08-01
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| Series: | Journal of Education, Health and Sport |
| Subjects: | |
| Online Access: | https://apcz.umk.pl/JEHS/article/view/26065 |
| _version_ | 1811303957838954496 |
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| author | Julita Poleszak Przemysław Szabat Marta Szabat Grzegorz Boreński Magdalena Wójcik Joanna Milanowska Konrad Rejdak |
| author_facet | Julita Poleszak Przemysław Szabat Marta Szabat Grzegorz Boreński Magdalena Wójcik Joanna Milanowska Konrad Rejdak |
| author_sort | Julita Poleszak |
| collection | DOAJ |
| description | 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: spinal muscular atrophy, nusinersen, zolgensma, reldesemtiv, branaplam, olesoxime.
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. |
| first_indexed | 2024-04-13T07:58:29Z |
| format | Article |
| id | doaj.art-dd96f26508d44271ad5aba3145c01147 |
| institution | Directory Open Access Journal |
| issn | 2391-8306 |
| language | English |
| last_indexed | 2024-04-13T07:58:29Z |
| publishDate | 2019-08-01 |
| publisher | Kazimierz Wielki University |
| record_format | Article |
| series | Journal of Education, Health and Sport |
| spelling | doaj.art-dd96f26508d44271ad5aba3145c011472022-12-22T02:55:21ZengKazimierz Wielki UniversityJournal of Education, Health and Sport2391-83062019-08-0198New prospects for the treatment of Spinal Muscular AtrophyJulita Poleszak0Przemysław Szabat1Marta Szabat2Grzegorz Boreński3Magdalena Wójcik4Joanna Milanowska5Konrad Rejdak6Students Scientific Society of Neurology, Medical University of LublinStudents Scientific Society of Neurology, Medical University of LublinStudent Science Club at the Department of Applied Psychology, Medical University of LublinStudent Science Club at the Department of Applied Psychology, Medical University of LublinStudent Science Club at the Department of Applied Psychology, Medical University of LublinDepartment of Applied Psychology, Medical University of LublinDepartment of Neurology, Medical University of LublinIntroduction: 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: spinal muscular atrophy, nusinersen, zolgensma, reldesemtiv, branaplam, olesoxime. 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.https://apcz.umk.pl/JEHS/article/view/26065nusinersenzolgensmareldesemtiv |
| spellingShingle | Julita Poleszak Przemysław Szabat Marta Szabat Grzegorz Boreński Magdalena Wójcik Joanna Milanowska Konrad Rejdak New prospects for the treatment of Spinal Muscular Atrophy Journal of Education, Health and Sport nusinersen zolgensma reldesemtiv |
| title | New prospects for the treatment of Spinal Muscular Atrophy |
| title_full | New prospects for the treatment of Spinal Muscular Atrophy |
| title_fullStr | New prospects for the treatment of Spinal Muscular Atrophy |
| title_full_unstemmed | New prospects for the treatment of Spinal Muscular Atrophy |
| title_short | New prospects for the treatment of Spinal Muscular Atrophy |
| title_sort | new prospects for the treatment of spinal muscular atrophy |
| topic | nusinersen zolgensma reldesemtiv |
| url | https://apcz.umk.pl/JEHS/article/view/26065 |
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