title: International guidelines for the management and treatment of Morquio A syndrome.
abstract: Morquio A syndrome (mucopolysaccharidosis IVA) is a lysosomal storage disorder associated with skeletal and joint abnormalities and significant non-skeletal manifestations including respiratory disease, spinal cord compression, cardiac disease, impaired vision, hearing loss, and dental problems. The clinical presentation, onset, severity and progression rate of clinical manifestations of Morquio A syndrome vary widely between patients. Because of the heterogeneous and progressive nature of the disease, the management of patients with Morquio A syndrome is challenging and requires a multidisciplinary approach, involving an array of specialists. The current paper presents international guidelines for the evaluation, treatment and symptom-based management of Morquio A syndrome. These guidelines were developed during two expert meetings by an international panel of specialists in pediatrics, genetics, orthopedics, pulmonology, cardiology, and anesthesia with extensive experience in managing Morquio A syndrome.
authors.affiliation: Salford Royal NHS Foundation Trust, Salford, UK.
authors.forename: Christian J
authors.initials: CJ
authors.lastname: Hendriksz
authors.forename: Kenneth I
authors.initials: KI
authors.lastname: Berger
authors.forename: Roberto
authors.initials: R
authors.lastname: Giugliani
authors.forename: Paul
authors.initials: P
authors.lastname: Harmatz
authors.forename: Christoph
authors.initials: C
authors.lastname: Kampmann
authors.forename: William G
authors.initials: WG
authors.lastname: Mackenzie
authors.forename: Julian
authors.initials: J
authors.lastname: Raiman
authors.forename: Martha Solano
authors.initials: MS
authors.lastname: Villarreal
authors.forename: Ravi
authors.initials: R
authors.lastname: Savarirayan
mesh_terms: D006801:Humans; D038622:Internationality; D009085:Mucopolysaccharidosis IV / Q000150:complications / Q000175:diagnosis / Q000000981:diagnostic imaging / Q000628:therapy*; D017410:Practice Guidelines as Topic; D011859:Radiography
keywords: diagnosis; disease management; guidelines; mucopolysaccharidosis IV; symptom assessment