Consensus concept of modern effective therapy for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a genetic orphan neuromuscular disease caused by a mutation in the DMD gene encoding the protein dystrophin. As a result of developing and progressive muscle damage and atrophy, children lose the ability to walk, develop respiratory and cardiac disorders. The core elem...

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Main Authors:	T. A. Gremyakova, S. B. Artemyeva, E. N. Baybarina, N. D. Vashakmadze, V. I. Guzeva, E. V. Gusakova, L. M. Kuzenkova, A. E. Lavrova, O. A. Lvova, S. V. Mikhaylova, L. P. Nazarenko, S. S. Nikitin, A. V. Polyakov, E. L. Dadali, A. G. Rumyantsev, G. E. Sakbaeva, V. M. Suslov, O. I. Gremyakova, A. A. Stepanov, N. I. Shakhovskaya
Format:	Article
Language:	Russian
Published:	ABV-press 2023-06-01
Series:	Нервно-мышечные болезни
Subjects:	duchenne muscular dystrophy recommendations dystrophin glucocorticosteroids vitamin d physical therapy gene therapy
Online Access:	https://nmb.abvpress.ru/jour/article/view/539
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Summary:	Duchenne muscular dystrophy is a genetic orphan neuromuscular disease caused by a mutation in the DMD gene encoding the protein dystrophin. As a result of developing and progressive muscle damage and atrophy, children lose the ability to walk, develop respiratory and cardiac disorders. The core elements of good care standards are early diagnosis, prevention and treatment of osteoporosis, daily physical therapy, regular rehabilitation, glucocorticosteroids, and control of heart and lung function. The clinical effect of new targeted pathogenetic therapies for Duchenne muscular dystrophy, restoring synthesis of full or truncated dystrophin, depend on their appropriate combination with existing standards of care.
ISSN:	2222-8721 2413-0443

Consensus concept of modern effective therapy for Duchenne muscular dystrophy

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