Poor bone health in Duchenne muscular dystrophy: a multifactorial problem beyond corticosteroids and loss of ambulation

Amelia Hurley-Novatny; David Chang; Katsuhiro Murakami; Ling Wang; Hongshuai Li

doi:10.3389/fendo.2024.1398050

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Poor bone health in Duchenne muscular dystrophy: a multifactorial problem beyond corticosteroids and loss of ambulation

Journal article

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Poor bone health in Duchenne muscular dystrophy: a multifactorial problem beyond corticosteroids and loss of ambulation

Amelia Hurley-Novatny, David Chang, Katsuhiro Murakami, Ling Wang and Hongshuai Li

Frontiers in endocrinology (Lausanne), Vol.15, 1398050

11/28/2024

DOI: 10.3389/fendo.2024.1398050

PMCID: PMC11634624

PMID: 39669499

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Abstract

Duchenne muscular dystrophy (DMD) is a progressive, fatal muscle wasting disease caused by X-linked mutations in the dystrophin gene. Alongside the characteristic muscle weakness, patients face a myriad of skeletal complications, including osteoporosis/osteopenia, high susceptibility to vertebral and long bone fractures, fat embolism post-fracture, scoliosis, and growth retardation. Those skeletal abnormalities significantly compromise quality of life and are sometimes life-threatening. These issues were traditionally attributed to loss of ambulation and chronic corticosteroid use, but recent investigations have unveiled a more intricate etiology. Factors such as vitamin D deficiency, hormonal imbalances, systemic inflammation, myokine release from dystrophic muscle, and vascular dysfunction are emerging as significant contributors as well. This expanded understanding illuminates the multifaceted pathogenesis underlying skeletal issues in DMD. Present therapeutic options are limited and lack specificity. Advancements in understanding the pathophysiology of bone complications in DMD will offer promising avenues for novel treatment modalities. In this review, we summarize the current understanding of factors contributing to bone problems in DMD and delineate contemporary and prospective multidisciplinary therapeutic approaches.

Adrenal Cortex Hormones - adverse effects

Adrenal Cortex Hormones - therapeutic use

Bone and Bones - metabolism

Bone Diseases, Metabolic - etiology

Humans

Muscular Dystrophy, Duchenne - complications

Muscular Dystrophy, Duchenne - physiopathology

Muscular Dystrophy, Duchenne - therapy

Osteoporosis - etiology

Details

Title: Subtitle: Poor bone health in Duchenne muscular dystrophy: a multifactorial problem beyond corticosteroids and loss of ambulation
Creators: Amelia Hurley-Novatny - University of Iowa
David Chang - University of Iowa
Katsuhiro Murakami - University of Iowa
Ling Wang - University of Iowa
Hongshuai Li - University of Iowa
Resource Type: Journal article
Publication Details: Frontiers in endocrinology (Lausanne), Vol.15, 1398050
DOI: 10.3389/fendo.2024.1398050
PMID: 39669499
PMCID: PMC11634624
NLM abbreviation: Front Endocrinol (Lausanne)
ISSN: 1664-2392
eISSN: 1664-2392
Publisher: FRONTIERS MEDIA SA
Grant note: NIH: 1R01AR076357, 1R01AR083398, 5R01HL098032 University of Iowa MSTP training: T32 GM139776
The author(s) declare that financial support was received for the research, authorship, and/or publication of this article. HL, LW, and AH-N receive research support from NIH grants 1R01AR076357 and 1R01AR083398. LW also receives research support from NIH grant 5R01HL098032. AH-N and DC are also supported by the University of Iowa MSTP training grant T32 GM139776.
Language: English
Date published: 11/28/2024
Academic Unit: Orthopedics and Rehabilitation; Radiation Oncology
Record Identifier: 9984757744602771

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