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Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study
Becker muscular dystrophy (BMD) is an inherited disorder due to deletions of the dystrophin gene that leads to muscle weakness. Effects of bone marrow mononuclear cell (BMMNC) transplantation in Muscular Dystrophy have shown to be safe and beneficial. We treated a 20-year-old male suffering from BMD...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
PAGEPress Publications, Pavia, Italy
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4821220/ https://www.ncbi.nlm.nih.gov/pubmed/27054018 http://dx.doi.org/10.4081/ejtm.2016.5522 |
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author | Sharma, Alok Sane, Hemangi Gokulchandra, Nandini Sharan, Rishabh Paranjape, Amruta Kulkarni, Pooja Yadav, Jayanti Badhe, Prerna |
author_facet | Sharma, Alok Sane, Hemangi Gokulchandra, Nandini Sharan, Rishabh Paranjape, Amruta Kulkarni, Pooja Yadav, Jayanti Badhe, Prerna |
author_sort | Sharma, Alok |
collection | PubMed |
description | Becker muscular dystrophy (BMD) is an inherited disorder due to deletions of the dystrophin gene that leads to muscle weakness. Effects of bone marrow mononuclear cell (BMMNC) transplantation in Muscular Dystrophy have shown to be safe and beneficial. We treated a 20-year-old male suffering from BMD with autologous BMMNC transplantation followed by multidisciplinary rehabilitation. He presented with muscle weakness and had difficulty in performing his activities. The BMMNCs were transplanted via intrathecal and intramuscular routes. The effects were measured on clinical and functional changes. Over 9 months, gradual improvement was noticed in muscle strength, respiratory functions and North Star Ambulatory Assessment Scale. Functional Independence Measure, Berg Balance Score, Brooke and Vignos Scale remained stable indicating halting of the progression. The case report suggests that cellular therapy combined with rehabilitation may have possibility of repairing and regenerating muscle fibers and decreasing the rate of progression of BMD. |
format | Online Article Text |
id | pubmed-4821220 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2016 |
publisher | PAGEPress Publications, Pavia, Italy |
record_format | MEDLINE/PubMed |
spelling | pubmed-48212202016-04-06 Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study Sharma, Alok Sane, Hemangi Gokulchandra, Nandini Sharan, Rishabh Paranjape, Amruta Kulkarni, Pooja Yadav, Jayanti Badhe, Prerna Eur J Transl Myol Original Article Becker muscular dystrophy (BMD) is an inherited disorder due to deletions of the dystrophin gene that leads to muscle weakness. Effects of bone marrow mononuclear cell (BMMNC) transplantation in Muscular Dystrophy have shown to be safe and beneficial. We treated a 20-year-old male suffering from BMD with autologous BMMNC transplantation followed by multidisciplinary rehabilitation. He presented with muscle weakness and had difficulty in performing his activities. The BMMNCs were transplanted via intrathecal and intramuscular routes. The effects were measured on clinical and functional changes. Over 9 months, gradual improvement was noticed in muscle strength, respiratory functions and North Star Ambulatory Assessment Scale. Functional Independence Measure, Berg Balance Score, Brooke and Vignos Scale remained stable indicating halting of the progression. The case report suggests that cellular therapy combined with rehabilitation may have possibility of repairing and regenerating muscle fibers and decreasing the rate of progression of BMD. PAGEPress Publications, Pavia, Italy 2016-03-31 /pmc/articles/PMC4821220/ /pubmed/27054018 http://dx.doi.org/10.4081/ejtm.2016.5522 Text en http://creativecommons.org/licenses/by-nc/3.0/ This article is distributed under the terms of the Creative Commons Attribution Noncommercial License (by-nc 3.0) which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and source are credited. |
spellingShingle | Original Article Sharma, Alok Sane, Hemangi Gokulchandra, Nandini Sharan, Rishabh Paranjape, Amruta Kulkarni, Pooja Yadav, Jayanti Badhe, Prerna Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study |
title | Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study |
title_full | Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study |
title_fullStr | Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study |
title_full_unstemmed | Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study |
title_short | Effect of Cellular Therapy in Progression of Becker’s Muscular Dystrophy: A Case Study |
title_sort | effect of cellular therapy in progression of becker’s muscular dystrophy: a case study |
topic | Original Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4821220/ https://www.ncbi.nlm.nih.gov/pubmed/27054018 http://dx.doi.org/10.4081/ejtm.2016.5522 |
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