Step-by-Step Double-Trouble OBAIRH and DMD Diagnosis in a One-Year-Old Boy

Author:

Shchagina Olga1,Kurilova Vera2,Zinina Elena1,Porubov Vyacheslav2,Efishova Svetlana2,Polyakov Aleksander1

Affiliation:

1. Research Centre for Medical Genetics, Moscow 115522, Russia

2. State Budgetary Institution of Health of the Perm Region “Regional Children’s Clinical Hospital”, Perm 614066, Russia

Abstract

We present a case of a combination of two rare hereditary disorders: obesity, adrenal insufficiency and red hair syndrome (OBAIRH) and Duchenne muscular dystrophy (DMD) in a boy. Both diseases were diagnosed during the first year of life. OBAIRH was suggested based on the ethnicity and family history of the patient, while DMD was based on an extreme increase in transaminase and CK (creatine kinase) levels during a biochemical analysis of his blood. The OBAIRH syndrome was caused by a pathogenic homozygous variant in the regulatory region of the POMC gene (NM_001035256.3): c.-71+1G>A, while DMD was caused by the de novo deletion of exons 38–45 of the DMD (NM_004006.3) gene (NC_000023.10:g.(?_32380941)(31950285_?)del).

Funder

Ministry of Science and Higher Education of the Russian Federation

Publisher

MDPI AG

Subject

Inorganic Chemistry,Organic Chemistry,Physical and Theoretical Chemistry,Computer Science Applications,Spectroscopy,Molecular Biology,General Medicine,Catalysis

Reference18 articles.

1. Components of Ethnic Identity of Tatars of Perm Territory (According to Spontaneous Texts);Khudiakova;Compon. Ethn. Identity Tatars Perm Territ.,2020

2. (2023, June 23). Rosstat—All-Russian Population Census, Available online: https://rosstat.gov.ru/vpn_popul.

3. Prognostic Factors, Disease Course, and Treatment Efficacy in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis;Weber;Muscle Nerve,2022

4. Drug Development Progress in Duchenne Muscular Dystrophy;Deng;Front. Pharmacol.,2022

5. Proopiomelanocortin Deficiency Diagnosed in Infancy in Two Boys and a Review of the Known Cases;Graves;J. Paediatr. Child Health,2021

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3