title: Enzyme replacement therapy in adult-onset glycogenosis II: is quantitative muscle MRI helpful?
abstract: Although it has been shown that muscle magnetic resonance imaging (MRI) improves the phenotypic characterization of patients with neuromuscular disorders and allows accurate quantification of muscle and adipose tissue distribution, to date quantitative MRI has not been used to assess the therapeutic response in clinical trials of neuromuscular diseases. We discuss quantitative MRI findings after a 6-month course of enzyme replacement therapy administered to nine patients with adult-onset glycogenosis II.
authors.affiliation: Neuroradiology Department, IRCCS "C. Mondino Institute of Neurology" Foundation, University of Pavia, Via Mondino 2, 27100 Pavia, Italy. anna.pichiecchio@mondino.it
authors.forename: A
authors.initials: A
authors.lastname: Pichiecchio
authors.forename: G U
authors.initials: GU
authors.lastname: Poloni
authors.forename: S
authors.initials: S
authors.lastname: Ravaglia
authors.forename: M
authors.initials: M
authors.lastname: Ponzio
authors.forename: G
authors.initials: G
authors.lastname: Germani
authors.forename: D
authors.initials: D
authors.lastname: Maranzana
authors.forename: A
authors.initials: A
authors.lastname: Costa
authors.forename: A
authors.initials: A
authors.lastname: Repetto
authors.forename: E
authors.initials: E
authors.lastname: Tavazzi
authors.forename: C
authors.initials: C
authors.lastname: Danesino
authors.forename: A
authors.initials: A
authors.lastname: Moglia
authors.forename: S
authors.initials: S
authors.lastname: Bastianello
mesh_terms: D017668:Age of Onset; D000368:Aged; D005260:Female; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy* / Q000473:pathology*; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods; D008297:Male; D008875:Middle Aged; D018482:Muscle, Skeletal / Q000473:pathology*; D011994:Recombinant Proteins / Q000627:therapeutic use; D000520:alpha-Glucosidases / Q000627:therapeutic use*
chemical_list: D011994:Recombinant Proteins; D000520:alpha-Glucosidases