Eteplirsen在男孩杜氏肌营养不良和镰状细胞性贫血中的应用。

IF 0.6 Q4 CLINICAL NEUROLOGY Case Reports in Neurology Pub Date : 2022-09-01 DOI:10.1159/000527358

Gregory M Aiello, Michael Stephen Cartwright

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引用次数: 1

摘要

Eteplirsen是一种用于治疗杜氏肌营养不良症(DMD)的反义寡核苷酸。对于有罕见合并症的个体，使用艾普生的安全性尚不清楚。我们提出的情况下，一个4岁的男孩与DMD外显子缺失可接受治疗的埃替普利森和合并症镰状细胞性贫血。他接受了3年的eteplirsen治疗，没有明显的不良反应，包括没有增加镰状细胞危象。

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Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia.

Eteplirsen is an antisense oligonucleotide used in the treatment of Duchenne muscular dystrophy (DMD). The safety of eteplirsen use in individuals with rare comorbid conditions is not known. We present the case of a 4-year-old boy with a DMD exon deletion amenable to treatment with eteplirsen and comorbid sickle cell anemia. He has received eteplirsen treatment for 3 years with no clear adverse effects, including no increase in sickle cell crises.

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来源期刊

Case Reports in Neurology Medicine-Neurology (clinical)

CiteScore

1.50

自引率

0.00%

发文量

审稿时长

14 weeks

期刊介绍： This new peer-reviewed online-only journal publishes original case reports covering the entire spectrum of neurology. Clinicians and researchers are given a tool to disseminate their personal experience to a wider public as well as to review interesting cases encountered by colleagues all over the world. To complement the contributions supplementary material is welcomed. The reports are searchable according to the key words supplied by the authors; it will thus be possible to search across the entire growing collection of case reports with universally used terms, further facilitating the retrieval of specific information. Following the open access principle, the entire contents can be retrieved at no charge, guaranteeing easy access to this valuable source of anecdotal information at all times.