What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models
Duchenne muscular dystrophy (DMD), the severest form of muscular dystrophy, is characterized by progressive muscle weakness with fatal outcomes most often before the fourth decade of life. Despite the recent addition of molecular treatments, DMD remains a disease without a cure, and the need persist...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-07-01
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| Series: | Biomedicines |
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| Online Access: | https://www.mdpi.com/2227-9059/11/7/2033 |
| _version_ | 1797590111631704064 |
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| author | Boel De Paepe |
| author_facet | Boel De Paepe |
| author_sort | Boel De Paepe |
| collection | DOAJ |
| description | Duchenne muscular dystrophy (DMD), the severest form of muscular dystrophy, is characterized by progressive muscle weakness with fatal outcomes most often before the fourth decade of life. Despite the recent addition of molecular treatments, DMD remains a disease without a cure, and the need persists for the development of supportive therapies aiming to help improve patients’ quality of life. This review focuses on the therapeutical potential of amino acid and derivative supplements, summarizing results obtained in preclinical studies in murine disease models. Several promising compounds have emerged, with L-arginine, N-acetylcysteine, and taurine featuring among the most intensively investigated. Their beneficial effects include reduced inflammatory, oxidative, fibrotic, and necrotic damage to skeletal muscle tissues. Improvement of muscle strength and endurance have been reported; however, mild side effects have also surfaced. More explorative, placebo-controlled and long-term clinical trials would need to be conducted in order to identify amino acid formulae that are safe and of true benefit to DMD patients. |
| first_indexed | 2024-03-11T01:16:48Z |
| format | Article |
| id | doaj.art-96765f52ed3941c8ac017c5c62f1e4ac |
| institution | Directory Open Access Journal |
| issn | 2227-9059 |
| language | English |
| last_indexed | 2024-03-11T01:16:48Z |
| publishDate | 2023-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomedicines |
| spelling | doaj.art-96765f52ed3941c8ac017c5c62f1e4ac2023-11-18T18:28:30ZengMDPI AGBiomedicines2227-90592023-07-01117203310.3390/biomedicines11072033What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse ModelsBoel De Paepe0Department of Neurology, Ghent University & Neuromuscular Reference Center, Ghent University Hospital, Route 830, Corneel Heymanslaan 10, 9000 Ghent, BelgiumDuchenne muscular dystrophy (DMD), the severest form of muscular dystrophy, is characterized by progressive muscle weakness with fatal outcomes most often before the fourth decade of life. Despite the recent addition of molecular treatments, DMD remains a disease without a cure, and the need persists for the development of supportive therapies aiming to help improve patients’ quality of life. This review focuses on the therapeutical potential of amino acid and derivative supplements, summarizing results obtained in preclinical studies in murine disease models. Several promising compounds have emerged, with L-arginine, N-acetylcysteine, and taurine featuring among the most intensively investigated. Their beneficial effects include reduced inflammatory, oxidative, fibrotic, and necrotic damage to skeletal muscle tissues. Improvement of muscle strength and endurance have been reported; however, mild side effects have also surfaced. More explorative, placebo-controlled and long-term clinical trials would need to be conducted in order to identify amino acid formulae that are safe and of true benefit to DMD patients.https://www.mdpi.com/2227-9059/11/7/2033N-acetylcysteineamino acid supplementsL-arginineDuchenne muscular dystrophymdx mouseosmolytes |
| spellingShingle | Boel De Paepe What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models Biomedicines N-acetylcysteine amino acid supplements L-arginine Duchenne muscular dystrophy mdx mouse osmolytes |
| title | What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models |
| title_full | What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models |
| title_fullStr | What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models |
| title_full_unstemmed | What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models |
| title_short | What Nutraceuticals Can Do for Duchenne Muscular Dystrophy: Lessons Learned from Amino Acid Supplementation in Mouse Models |
| title_sort | what nutraceuticals can do for duchenne muscular dystrophy lessons learned from amino acid supplementation in mouse models |
| topic | N-acetylcysteine amino acid supplements L-arginine Duchenne muscular dystrophy mdx mouse osmolytes |
| url | https://www.mdpi.com/2227-9059/11/7/2033 |
| work_keys_str_mv | AT boeldepaepe whatnutraceuticalscandoforduchennemusculardystrophylessonslearnedfromaminoacidsupplementationinmousemodels |