title: Sixty six-month follow-up of muscle power and respiratory function in a case with adult-type Pompe disease treated with enzyme replacement therapy.
abstract: We report a patient with adult-type Pompe disease treated with enzyme replacement therapy (ERT) for 5.5 years. We evaluated pulmonary function and muscle strength using 6-minute walk test, manual muscle test, and dynamometer-based measurement. The long-term ERT resulted in a substantial improvement in the pulmonary function and a possible stabilization followed by mild deterioration in muscle power measured by dynamometer and 6-minute walk test. Our data may rationalize the long-term use of ERT for adult-type Pompe disease in terms of maintaining pulmonary function.
authors.affiliation: Departments of *Neurology and †Rehabilitation, Kyoto Prefectural University of Medicine, Kyoto, Japan; and ‡Kyoto Prefectural Support Center of Rehabilitation, Kyoto, Japan.
authors.forename: Reina
authors.initials: R
authors.lastname: Isayama
authors.forename: Kensuke
authors.initials: K
authors.lastname: Shiga
authors.forename: Kazuya
authors.initials: K
authors.lastname: Seo
authors.forename: Yumiko
authors.initials: Y
authors.lastname: Azuma
authors.forename: Yasukiyo
authors.initials: Y
authors.lastname: Araki
authors.forename: Ai
authors.initials: A
authors.lastname: Hamano
authors.forename: Hedesato
authors.initials: H
authors.lastname: Takezawa
authors.forename: Nagato
authors.initials: N
authors.lastname: Kuriyama
authors.forename: Nobuo
authors.initials: N
authors.lastname: Takezawa
authors.forename: Toshiki
authors.initials: T
authors.lastname: Mizuno
authors.forename: Masanori
authors.initials: M
authors.lastname: Nakagawa
mesh_terms: D000328:Adult; D018450:Disease Progression; D056947:Enzyme Replacement Therapy; D005500:Follow-Up Studies; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy* / Q000503:physiopathology*; D006801:Humans; D007074:Immunoglobulin G / Q000032:analysis; D008297:Male; D053580:Muscle Strength; D018482:Muscle, Skeletal / Q000503:physiopathology*; D026741:Physical Therapy Modalities; D012119:Respiration; D012129:Respiratory Function Tests; D000520:alpha-Glucosidases / Q000627:therapeutic use
chemical_list: D007074:Immunoglobulin G; D000520:alpha-Glucosidases