Reduced serum myostatin concentrations associated with genetic muscle disease progression

Myostatin is a highly conserved protein secreted primarily from skeletal muscle that can potently suppress muscle growth. This ability to regulate skeletal muscle mass has sparked intense interest in the development of anti-myostatin therapies for a wide array of muscle disorders including sarcopeni...

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Bibliographic Details
Published in:Journal of neurology 2017-03, Vol.264 (3), p.541-553
Main Authors: Burch, Peter M., Pogoryelova, Oksana, Palandra, Joe, Goldstein, Richard, Bennett, Donald, Fitz, Lori, Guglieri, Michela, Bettolo, Chiara Marini, Straub, Volker, Evangelista, Teresinha, Neubert, Hendrik, Lochmüller, Hanns, Morris, Carl
Format: Article
Language:English
Subjects:
Adolescent
Adult
Aged
Aged, 80 and over
Animals
Biomarkers
Biomarkers - blood
Blood Chemical Analysis
Child
Child, Preschool
Cohort Studies
Disease
Disease Progression
Female
Humans
Kinases
Male
Medicine
Medicine & Public Health
Mice, Inbred C57BL
Mice, Transgenic
Middle Aged
Muscular dystrophy
Musculoskeletal system
Mutation
Myostatin - blood
Neurology
Neuromuscular diseases
Neuromuscular Diseases - blood
Neuromuscular Diseases - genetics
Neuroradiology
Neurosciences
Original Communication
Patients
Proteins
R&D
Research & development
Sarcopenia
Young Adult
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Summary:Myostatin is a highly conserved protein secreted primarily from skeletal muscle that can potently suppress muscle growth. This ability to regulate skeletal muscle mass has sparked intense interest in the development of anti-myostatin therapies for a wide array of muscle disorders including sarcopenia, cachexia and genetic neuromuscular diseases. While a number of studies have examined the circulating myostatin concentrations in healthy and sarcopenic populations, very little data are available from inherited muscle disease patients. Here, we have measured the myostatin concentration in serum from seven genetic neuromuscular disorder patient populations using immunoaffinity LC–MS/MS. Average serum concentrations of myostatin in all seven muscle disease patient groups were significantly less than those measured in healthy controls. Furthermore, circulating myostatin concentrations correlated with clinical measures of disease progression for five of the muscle disease patient populations. These findings greatly expand the understanding of myostatin in neuromuscular disease and suggest its potential utility as a biomarker of disease progression.
ISSN:0340-5354
1432-1459
DOI:10.1007/s00415-016-8379-6