{"title":"Advances in Therapeutics—Enzyme Replacement Therapy: Long-Term Evaluation and New Approaches","authors":"Robin Lachmann","doi":"10.1002/jimd.70166","DOIUrl":null,"url":null,"abstract":"<div>\n \n <p>The first enzyme replacement therapy (ERT) for a lysosomal storage disorder was marketed more than 30 years ago. We now have licensed products for 12 different conditions, and for a number of conditions, up to three different preparations are commercially available. These products were licensed on the basis of their ability to achieve pre-defined endpoints in clinical trials, but many have now been in clinical use for decades, and large amounts of real-world evidence have been generated. This has shown that not all treatments are equally effective and that there is still much unmet need, even for patients who have been treated for many years. Second-generation enzyme therapies are now being designed that will hopefully lead to improved clearance from currently hard-to-access cells and tissues, hence improving long-term clinical outcomes.</p>\n </div>","PeriodicalId":16281,"journal":{"name":"Journal of Inherited Metabolic Disease","volume":"49 2","pages":""},"PeriodicalIF":3.8000,"publicationDate":"2026-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Inherited Metabolic Disease","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/jimd.70166","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
引用次数: 0
Abstract
The first enzyme replacement therapy (ERT) for a lysosomal storage disorder was marketed more than 30 years ago. We now have licensed products for 12 different conditions, and for a number of conditions, up to three different preparations are commercially available. These products were licensed on the basis of their ability to achieve pre-defined endpoints in clinical trials, but many have now been in clinical use for decades, and large amounts of real-world evidence have been generated. This has shown that not all treatments are equally effective and that there is still much unmet need, even for patients who have been treated for many years. Second-generation enzyme therapies are now being designed that will hopefully lead to improved clearance from currently hard-to-access cells and tissues, hence improving long-term clinical outcomes.
期刊介绍：
The Journal of Inherited Metabolic Disease (JIMD) is the official journal of the Society for the Study of Inborn Errors of Metabolism (SSIEM). By enhancing communication between workers in the field throughout the world, the JIMD aims to improve the management and understanding of inherited metabolic disorders. It publishes results of original research and new or important observations pertaining to any aspect of inherited metabolic disease in humans and higher animals. This includes clinical (medical, dental and veterinary), biochemical, genetic (including cytogenetic, molecular and population genetic), experimental (including cell biological), methodological, theoretical, epidemiological, ethical and counselling aspects. The JIMD also reviews important new developments or controversial issues relating to metabolic disorders and publishes reviews and short reports arising from the Society''s annual symposia. A distinction is made between peer-reviewed scientific material that is selected because of its significance for other professionals in the field and non-peer- reviewed material that aims to be important, controversial, interesting or entertaining (“Extras”).
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