• T. Gremyakova
  • S. Artemyeva
  • E. Baybarina
  • N. Vashakmadze
  • V. Guzeva
  • E. Gusakova
  • L. Kuzenkova
  • A. Lavrova
  • O. Lvova
  • S. Mikhaylova
  • L. Nazarenko
  • S. Nikitin
  • A. Polyakov
  • E. l. Dadali
  • A. Rumyantsev
  • G. Sakbaeva
  • V. Suslov
  • O. Gremyakova
  • A. Stepanov
  • N. Shakhovskaya
Duchenne muscular dystrophy is a genetic orphan neuromuscular disease caused by a mutation in the DMD gene encod‑ ing the protein dystrophin. As a result of developing and progressive muscle damage and atrophy, childrenlose the abil‑ ity 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 andlung 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.
Translated title of the contributionConsensus concept of modern effective therapy for Duchenne muscular dystrophy
Original languageRussian
Pages (from-to)10-19
Number of pages10
JournalNeuromuscular Diseases
Volume13
Issue number2
DOIs
Publication statusPublished - 2023

    ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

ID: 43274094