234th ENMC International Workshop: Chaperone dysfunction in muscle disease Naarden, The Netherlands, 8–10 December 2017

Conrad C. Weihl, Bjarne Udd, Michael Hanna, ENMC workshop study group

Research output: Contribution to journalEditorialOtherpeer-review

1 Citation (Scopus)

Abstract

Twenty participants from Australia, Belgium, Denmark, Finland, France, Germany, Italy, Israel, The Netherlands, Sweden, UK and the USA met in Naarden, The Netherlands from December 8th–10th, 2017. This group included clinicians, clinical trialists, basic scientists, industry and patient representatives. Patients were represented by the Muscular Dystrophy Association and Alexander's Way (BAG3 associated myopathy). The goals were to discuss the role of protein chaperones in normal muscle function, muscle disease and future therapies. Protein chaperones are a class of proteins that participate in facilitating the proper folding and assembly of protein complexes. Thus, chaperones are essential for the development and maintenance of skeletal muscle. Specifically this large group of proteins ensures that other proteins (client proteins) maintain proper structure and function or if needed, facilitates their degradation via proteolytic pathways. Chaperone dysfunction is responsible for many rare hereditary myopathies; thus correcting chaperone function may be a therapeutic option.
Original languageEnglish
Pages (from-to)1022–1030
Number of pages9
JournalNeuromuscular Disorders
DOIs
Publication statusPublished - Dec 2018
Externally publishedYes

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