Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments

被引:0
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作者
Kazuki Sawamoto
Molly Stapleton
Carlos J. Alméciga-Díaz
Angela J. Espejo-Mojica
Juan Camilo Losada
Diego A. Suarez
Shunji Tomatsu
机构
[1] Nemours/Alfred I. duPont Hospital for Children,Nemours Biomedical Research
[2] University of Delaware,Department of Biological Sciences
[3] Pontificia Universidad Javeriana,Institute for the Study of Inborn Errors of Metabolism, Faculty of Science
[4] Universidad Nacional de Colombia,Facultad de Medicina
[5] Shimane University,Department of Pediatrics
[6] Gifu University,Department of Pediatrics
[7] Thomas Jefferson University,undefined
来源
Drugs | 2019年 / 79卷
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摘要
Mucopolysaccharidoses (MPS) are inborn errors of metabolism produced by a deficiency of one of the enzymes involved in the degradation of glycosaminoglycans (GAGs). Although taken separately, each type is rare. As a group, MPS are relatively frequent, with an overall estimated incidence of around 1 in 20,000–25,000 births. Development of therapeutic options for MPS, including hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT), has modified the natural history of many MPS types. In spite of the improvement in some tissues and organs, significant challenges remain unsolved, including blood–brain barrier (BBB) penetration and treatment of lesions in avascular cartilage, heart valves, and corneas. Newer approaches, such as intrathecal ERT, ERT with fusion proteins to cross the BBB, gene therapy, substrate reduction therapy (SRT), chaperone therapy, and some combination of these strategies may provide better outcomes for MPS patients in the near future. As early diagnosis and early treatment are imperative to improve therapeutic efficacy, the inclusion of MPS in newborn screening programs should enhance the potential impact of treatment in reducing the morbidity associated with MPS diseases. In this review, we evaluate available treatments, including ERT and HSCT, and future treatments, such as gene therapy, SRT, and chaperone therapy, and describe the advantages and disadvantages. We also assess the current clinical endpoints and biomarkers used in clinical trials.
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页码:1103 / 1134
页数:31
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