Cargando…

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...

Descripción completa

Detalles Bibliográficos
Autores principales: Sharma, Alok, Sane, Hemangi, Gokulchandra, Nandini, Sharan, Rishabh, Paranjape, Amruta, Kulkarni, Pooja, Yadav, Jayanti, Badhe, Prerna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: PAGEPress Publications, Pavia, Italy 2016
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
_version_ 1782425544324808704
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
work_keys_str_mv AT sharmaalok effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT sanehemangi effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT gokulchandranandini effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT sharanrishabh effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT paranjapeamruta effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT kulkarnipooja effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT yadavjayanti effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy
AT badheprerna effectofcellulartherapyinprogressionofbeckersmusculardystrophyacasestudy