{"title":"Gene Therapy: A New Hope in Sickle Cell Disease Treatment.","authors":"Margaret Christy, Beth Fisher","doi":"10.1016/j.pedhc.2024.07.007","DOIUrl":null,"url":null,"abstract":"<p><p>Sickle cell disease (SCD) is a lifelong disease requiring expensive treatment for management and limited curative options until the last few years. Gene therapy has emerged as a curative option for SCD, with two approved therapies available to SCD patients aged ≥ 12 years. Consideration must be considered regarding the ethics, efficacy, management requirements, education, and counseling needs of patients and their parents. Current and future practices will need to advocate for improved access and affordability of this specialized care and address the unknown and less defined areas of gene therapy pertaining to SCD through research.</p>","PeriodicalId":50094,"journal":{"name":"Journal of Pediatric Health Care","volume":"39 1","pages":"122-129"},"PeriodicalIF":2.5000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Pediatric Health Care","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.pedhc.2024.07.007","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEALTH POLICY & SERVICES","Score":null,"Total":0}
引用次数: 0
Abstract
Sickle cell disease (SCD) is a lifelong disease requiring expensive treatment for management and limited curative options until the last few years. Gene therapy has emerged as a curative option for SCD, with two approved therapies available to SCD patients aged ≥ 12 years. Consideration must be considered regarding the ethics, efficacy, management requirements, education, and counseling needs of patients and their parents. Current and future practices will need to advocate for improved access and affordability of this specialized care and address the unknown and less defined areas of gene therapy pertaining to SCD through research.
期刊介绍：
The Journal of Pediatric Health Care, the official journal of the National Association of Pediatric Nurse Practitioners, provides scholarly clinical information and research regarding primary, acute and specialty health care for children of newborn age through young adulthood within a family-centered context. The Journal disseminates multidisciplinary perspectives on evidence-based practice and emerging policy, advocacy and educational issues that are of importance to all healthcare professionals caring for children and their families.
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