Pediatrics · 2009
Multidisciplinary management of Hunter syndrome.
СтатьяОбзорНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Muenzer Joseph, Beck M, Eng C M, Escolar M L, Giugliani R, Guffon N H, Harmatz P, Kamin W, Kampmann C, Koseoglu S T, Link B, Martin R A, Molter D W, Muñoz Rojas M V, Ogilvie J W, Parini R, Ramaswami U, Scarpa M, Schwartz I V, Wood R E, Wraith E
Аннотация
Hunter syndrome is a rare, X-linked disorder caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase. In the absence of sufficient enzyme activity, glycosaminoglycans accumulate in the lysosomes of many tissues and organs and contribute to the multisystem, progressive pathologies seen in Hunter syndrome. The nervous, cardiovascular, respiratory, and musculoskeletal systems can be involved in individuals with Hunter syndrome. Although the management of some clinical problems associated with the disease may seem routine, the management is typically complex and requires the physician to be aware of the special issues surrounding the patient with Hunter syndrome, and a multidisciplinary approach should be taken. Subspecialties such as otorhinolaryngology, neurosurgery, orthopedics, cardiology, anesthesiology, pulmonology, and neurodevelopment will all have a role in management, as will specialty areas such as physiotherapy, audiology, and others. The important management topics are discussed in this review, and the use of enzyme-replacement therapy with recombinant human iduronate-2-sulfatase as a specific treatment for Hunter syndrome is presented.
Предметные рубрики MeSH
AdolescentAdultChildChild, PreschoolCombined Modality TherapyCooperative BehaviorEnzyme Replacement TherapyGenotypeHematopoietic Stem Cell TransplantationHumansIduronate SulfataseInfantInfant, NewbornInfusions, IntravenousInterdisciplinary CommunicationMaleMucopolysaccharidosis IIPatient Care TeamPhenotypeRandomized Controlled Trials as TopicRecombinant ProteinsYoung Adult