Duchenne Muscular Dystrophy: Modern Approaches in Patient Management

Duchenne muscular dystrophy is one of the most common forms of childhood muscular dystrophies. Its incidence is 1 in 3.5–6 thousand newborn boys according to various sources. The disease is caused by the mutation in the DMD gene coding the dystrophin protein, it leads to the dystrophin absence or ma...

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Main Authors: Inga V. Anisimova, Svetlana B. Artemyeva, Elena D. Belousova, Nato D. Vashakmadze, Dmitriy V. Vlodavets, Tatiana A. Gremyakova, Olga S. Groznova, Valentina I. Guzeva, Elena V. Gusakova, Lyudmila M. Kuzenkova, Alexey L. Kurenkov, Sergey I. Kutsev, Svetlana V. Mikhaylova, Lyudmila P. Nazarenko, Sergey S. Nikitin, Artem Yu. Novikov, Tatiana V. Podkletnova, Elena V. Polevichenko, Alexander V. Polyakov, Gennady G. Prokopyev, Dmitry I. Rudenko, Svetlana A. Repina, Evgeniia V. Romanenko, Sergey O. Ryabykh, Gul’zhan E. Sakbaeva, Elena Yu. Sapego, Liliia R. Selimzyanova, Andrey A. Stepanov, Dmitry M. Subbotin, Vasiliy M. Suslov, Elena V. Tozliyan, Dmirty A. Feklistov, Nadezhda I. Shakhovskaya, Ekaterina V. Shreder
Format: Article
Language:Russian
Published: Union of pediatricians of Russia 2023-11-01
Series:Педиатрическая фармакология
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Online Access:https://www.pedpharma.ru/jour/article/view/2348
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author Inga V. Anisimova
Svetlana B. Artemyeva
Elena D. Belousova
Nato D. Vashakmadze
Dmitriy V. Vlodavets
Tatiana A. Gremyakova
Olga S. Groznova
Valentina I. Guzeva
Elena V. Gusakova
Lyudmila M. Kuzenkova
Alexey L. Kurenkov
Sergey I. Kutsev
Svetlana V. Mikhaylova
Lyudmila P. Nazarenko
Sergey S. Nikitin
Artem Yu. Novikov
Tatiana V. Podkletnova
Elena V. Polevichenko
Alexander V. Polyakov
Gennady G. Prokopyev
Dmitry I. Rudenko
Svetlana A. Repina
Evgeniia V. Romanenko
Sergey O. Ryabykh
Gul’zhan E. Sakbaeva
Elena Yu. Sapego
Liliia R. Selimzyanova
Andrey A. Stepanov
Dmitry M. Subbotin
Vasiliy M. Suslov
Elena V. Tozliyan
Dmirty A. Feklistov
Nadezhda I. Shakhovskaya
Ekaterina V. Shreder
author_facet Inga V. Anisimova
Svetlana B. Artemyeva
Elena D. Belousova
Nato D. Vashakmadze
Dmitriy V. Vlodavets
Tatiana A. Gremyakova
Olga S. Groznova
Valentina I. Guzeva
Elena V. Gusakova
Lyudmila M. Kuzenkova
Alexey L. Kurenkov
Sergey I. Kutsev
Svetlana V. Mikhaylova
Lyudmila P. Nazarenko
Sergey S. Nikitin
Artem Yu. Novikov
Tatiana V. Podkletnova
Elena V. Polevichenko
Alexander V. Polyakov
Gennady G. Prokopyev
Dmitry I. Rudenko
Svetlana A. Repina
Evgeniia V. Romanenko
Sergey O. Ryabykh
Gul’zhan E. Sakbaeva
Elena Yu. Sapego
Liliia R. Selimzyanova
Andrey A. Stepanov
Dmitry M. Subbotin
Vasiliy M. Suslov
Elena V. Tozliyan
Dmirty A. Feklistov
Nadezhda I. Shakhovskaya
Ekaterina V. Shreder
author_sort Inga V. Anisimova
collection DOAJ
description Duchenne muscular dystrophy is one of the most common forms of childhood muscular dystrophies. Its incidence is 1 in 3.5–6 thousand newborn boys according to various sources. The disease is caused by the mutation in the DMD gene coding the dystrophin protein, it leads to the dystrophin absence or malfunction. The disease is characterized by proximal muscle weakness and gastrocnemius muscles pseudohypertrophy. In average, patients lose the ability to walk by themselves by the age of 11 and become nonambulatory. The authors have present modern epidemiological data and etiopathogenesis features of Duchenne muscular dystrophy, and have described clinical signs of different disease stages. The algorithm and key points of differential diagnosis are indicated. Special attention was given to the patients’ management: pathogenetic treatment and rehabilitation of pediatric patients.
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publisher Union of pediatricians of Russia
record_format Article
series Педиатрическая фармакология
spelling doaj-art-376b5dfeb86d48aa82f78ab3af2f103f2025-08-20T02:54:11ZrusUnion of pediatricians of RussiaПедиатрическая фармакология1727-57762500-30892023-11-0120542745310.15690/pf.v20i5.26152020Duchenne Muscular Dystrophy: Modern Approaches in Patient ManagementInga V. Anisimova0Svetlana B. Artemyeva1Elena D. Belousova2Nato D. Vashakmadze3Dmitriy V. Vlodavets4Tatiana A. Gremyakova5Olga S. Groznova6Valentina I. Guzeva7Elena V. Gusakova8Lyudmila M. Kuzenkova9Alexey L. Kurenkov10Sergey I. Kutsev11Svetlana V. Mikhaylova12Lyudmila P. Nazarenko13Sergey S. Nikitin14Artem Yu. Novikov15Tatiana V. Podkletnova16Elena V. Polevichenko17Alexander V. Polyakov18Gennady G. Prokopyev19Dmitry I. Rudenko20Svetlana A. Repina21Evgeniia V. Romanenko22Sergey O. Ryabykh23Gul’zhan E. Sakbaeva24Elena Yu. Sapego25Liliia R. Selimzyanova26Andrey A. Stepanov27Dmitry M. Subbotin28Vasiliy M. Suslov29Elena V. Tozliyan30Dmirty A. Feklistov31Nadezhda I. Shakhovskaya32Ekaterina V. Shreder33Research Centre for Medical GeneticsVeltischev Research and Clinical Institute for PediatricsVeltischev Research and Clinical Institute for PediatricsResearch Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical UniversityVeltischev Research and Clinical Institute for PediatricsInternational Advisory Council of the DMD Patient’s Community; Charity Fund “Gordey”Veltischev Research and Clinical Institute for PediatricsSaint-Petersburg State Pediatric Medical UniversityCentral Clinical Hospital with Outpatient Health Center of the office of PresidentNational Medical Research Center of Children’s HealthNational Medical Research Center of Children’s HealthResearch Centre for Medical GeneticsRussian Children’s Clinical HospitalResearch Institute of Medical GeneticsResearch Centre for Medical Genetics“Rassvet” ClinicNational Medical Research Center of Children’s HealthPirogov Russian National Research Medical UniversityResearch Centre for Medical GeneticsPirogov Russian National Research Medical University; V.F. Voyno-Yasenetsky Scientific and Practical Center of Specialized Medical Care for ChildrenCity Multidisciplinary Hospital №2 of Pavlov First Saint Petersburg State Medical UniversityResearch Centre for Medical GeneticsLighthouse Charity FoundationNational Ilizarov Medical Research Centre for Traumatology and OrtopaedicsCentral Clinical Hospital with Outpatient Health Center of the office of PresidentRegional Children’s Clinical HospitalResearch Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery; Sechenov First Moscow State Medical UniversityCentral Clinical Hospital with Outpatient Health Center of the office of PresidentResearch Centre for Medical GeneticsSaint-Petersburg State Pediatric Medical UniversityVeltischev Research and Clinical Institute for PediatricsVeltischev Research and Clinical Institute for PediatricsPsychoneurological Hospital for Children with Disturbance of the Central Nervous System with Mental DisordersCentral Clinical Hospital with Outpatient Health Center of the office of PresidentDuchenne muscular dystrophy is one of the most common forms of childhood muscular dystrophies. Its incidence is 1 in 3.5–6 thousand newborn boys according to various sources. The disease is caused by the mutation in the DMD gene coding the dystrophin protein, it leads to the dystrophin absence or malfunction. The disease is characterized by proximal muscle weakness and gastrocnemius muscles pseudohypertrophy. In average, patients lose the ability to walk by themselves by the age of 11 and become nonambulatory. The authors have present modern epidemiological data and etiopathogenesis features of Duchenne muscular dystrophy, and have described clinical signs of different disease stages. The algorithm and key points of differential diagnosis are indicated. Special attention was given to the patients’ management: pathogenetic treatment and rehabilitation of pediatric patients.https://www.pedpharma.ru/jour/article/view/2348duchenne myodystrophyhereditary diseasescardiomyopathiespathogenetic therapyrehabilitationchildren
spellingShingle Inga V. Anisimova
Svetlana B. Artemyeva
Elena D. Belousova
Nato D. Vashakmadze
Dmitriy V. Vlodavets
Tatiana A. Gremyakova
Olga S. Groznova
Valentina I. Guzeva
Elena V. Gusakova
Lyudmila M. Kuzenkova
Alexey L. Kurenkov
Sergey I. Kutsev
Svetlana V. Mikhaylova
Lyudmila P. Nazarenko
Sergey S. Nikitin
Artem Yu. Novikov
Tatiana V. Podkletnova
Elena V. Polevichenko
Alexander V. Polyakov
Gennady G. Prokopyev
Dmitry I. Rudenko
Svetlana A. Repina
Evgeniia V. Romanenko
Sergey O. Ryabykh
Gul’zhan E. Sakbaeva
Elena Yu. Sapego
Liliia R. Selimzyanova
Andrey A. Stepanov
Dmitry M. Subbotin
Vasiliy M. Suslov
Elena V. Tozliyan
Dmirty A. Feklistov
Nadezhda I. Shakhovskaya
Ekaterina V. Shreder
Duchenne Muscular Dystrophy: Modern Approaches in Patient Management
Педиатрическая фармакология
duchenne myodystrophy
hereditary diseases
cardiomyopathies
pathogenetic therapy
rehabilitation
children
title Duchenne Muscular Dystrophy: Modern Approaches in Patient Management
title_full Duchenne Muscular Dystrophy: Modern Approaches in Patient Management
title_fullStr Duchenne Muscular Dystrophy: Modern Approaches in Patient Management
title_full_unstemmed Duchenne Muscular Dystrophy: Modern Approaches in Patient Management
title_short Duchenne Muscular Dystrophy: Modern Approaches in Patient Management
title_sort duchenne muscular dystrophy modern approaches in patient management
topic duchenne myodystrophy
hereditary diseases
cardiomyopathies
pathogenetic therapy
rehabilitation
children
url https://www.pedpharma.ru/jour/article/view/2348
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