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Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review

Facioscapulohumeral muscular dystrophy (FSHD) is the third most common type of muscular dystrophy. This disease presents as a slowly progressive asymmetric muscle weakness that involves the facial, scapular, and upper arm muscles mainly. Currently, there is no established consensus on this disease t...

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Autores principales: Aguirre, Alex S, Astudillo Moncayo, Olga M, Mosquera, Johanna, Muyolema Arce, Veronica E, Gallegos, Camila, Ortiz, Juan Fernando, Andrade, Andres F, Oña, Sebastian, Buj, Maja J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cureus 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10316996/
https://www.ncbi.nlm.nih.gov/pubmed/37404420
http://dx.doi.org/10.7759/cureus.39903
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author Aguirre, Alex S
Astudillo Moncayo, Olga M
Mosquera, Johanna
Muyolema Arce, Veronica E
Gallegos, Camila
Ortiz, Juan Fernando
Andrade, Andres F
Oña, Sebastian
Buj, Maja J
author_facet Aguirre, Alex S
Astudillo Moncayo, Olga M
Mosquera, Johanna
Muyolema Arce, Veronica E
Gallegos, Camila
Ortiz, Juan Fernando
Andrade, Andres F
Oña, Sebastian
Buj, Maja J
author_sort Aguirre, Alex S
collection PubMed
description Facioscapulohumeral muscular dystrophy (FSHD) is the third most common type of muscular dystrophy. This disease presents as a slowly progressive asymmetric muscle weakness that involves the facial, scapular, and upper arm muscles mainly. Currently, there is no established consensus on this disease treatment in terms of medications. We assessed the response to the treatment of the drugs utilized in clinical trials by performing a systematic literature review in English using the preferred reporting items for systematic reviews (PRISMA) and meta-analyses. We only used human clinical trials in patients diagnosed with FSHD that received consistent pharmacological treatment. We included 11 clinical trials that fulfilled our criteria. We concluded that albuterol had statistically significant results in three out of four clinical trials, with improved elbow flexors muscle strength. Vitamin C, vitamin E, zinc gluconate, and selenomethionine showed significant improvement in the maximal voluntary contraction and endurance limit time of quadriceps muscle. At the same time, diltiazem and MYO-029 demonstrate no improvement in function, strength, or muscle mass. Losmapimod, currently in phase I of the ReDUX4 trial, showed promising results. Peradventure, more clinical trials are still needed to address this subject. Nevertheless, this review provides a clear and concise update on the treatment for this disease.
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spelling pubmed-103169962023-07-04 Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review Aguirre, Alex S Astudillo Moncayo, Olga M Mosquera, Johanna Muyolema Arce, Veronica E Gallegos, Camila Ortiz, Juan Fernando Andrade, Andres F Oña, Sebastian Buj, Maja J Cureus Neurology Facioscapulohumeral muscular dystrophy (FSHD) is the third most common type of muscular dystrophy. This disease presents as a slowly progressive asymmetric muscle weakness that involves the facial, scapular, and upper arm muscles mainly. Currently, there is no established consensus on this disease treatment in terms of medications. We assessed the response to the treatment of the drugs utilized in clinical trials by performing a systematic literature review in English using the preferred reporting items for systematic reviews (PRISMA) and meta-analyses. We only used human clinical trials in patients diagnosed with FSHD that received consistent pharmacological treatment. We included 11 clinical trials that fulfilled our criteria. We concluded that albuterol had statistically significant results in three out of four clinical trials, with improved elbow flexors muscle strength. Vitamin C, vitamin E, zinc gluconate, and selenomethionine showed significant improvement in the maximal voluntary contraction and endurance limit time of quadriceps muscle. At the same time, diltiazem and MYO-029 demonstrate no improvement in function, strength, or muscle mass. Losmapimod, currently in phase I of the ReDUX4 trial, showed promising results. Peradventure, more clinical trials are still needed to address this subject. Nevertheless, this review provides a clear and concise update on the treatment for this disease. Cureus 2023-06-03 /pmc/articles/PMC10316996/ /pubmed/37404420 http://dx.doi.org/10.7759/cureus.39903 Text en Copyright © 2023, Aguirre et al. https://creativecommons.org/licenses/by/3.0/This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Neurology
Aguirre, Alex S
Astudillo Moncayo, Olga M
Mosquera, Johanna
Muyolema Arce, Veronica E
Gallegos, Camila
Ortiz, Juan Fernando
Andrade, Andres F
Oña, Sebastian
Buj, Maja J
Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review
title Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review
title_full Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review
title_fullStr Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review
title_full_unstemmed Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review
title_short Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review
title_sort treatment of facioscapulohumeral muscular dystrophy (fshd): a systematic review
topic Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10316996/
https://www.ncbi.nlm.nih.gov/pubmed/37404420
http://dx.doi.org/10.7759/cureus.39903
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