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Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone

Title: Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone
Authors: Sbrocchi AM; Kinnett K; Lautatzis M-E; Mcmillan HJ; Selby KA; Veerapandiyan A; Weber DR; Apkon S; Bharucha-Goebel DX; Bharill S; Bansal S; Clemens PR; Fiscaletti M; Halloun R; Lam C; Merchant N; Mcadam L; Nasomyont N; Nicolau S; Ochoa Molina MF; Phung K; Rutter MM; Scavina M; Surampudi PN; Tamaroff J; Tian C; Ward LM; Wood CL; Wong SC; Ahmet A; Furlong P; McMillan HJ; Manesh RP; Wang M; Schrader R; Lam CKL; Babalola F; Crane JL; Sorbara JC; Benjamin R; Molina MFO; Renthal NE; Appel AM; Vargas G; Bratland LE; Truba N; Hoskin J; Werth C; Moeschen P; Zeitler P; Lewis C
Source: Journal of the Endocrine Society, 1 February 2026
Publisher Information: Endocrine Society
Publication Year: 2026
Collection: Newcastle University Library ePrints Service
Description: © 2025 The Author(s). Published by Oxford University Press on behalf of the Endocrine Society.Adrenal suppression is an iatrogenic form of adrenal insufficiency that occurs secondary to exogenous glucocorticoids (GCs) and is a documented cause of premature mortality among individuals with Duchenne muscular dystrophy (DMD). Adrenal suppression in DMD necessitates awareness and careful management, given that GCs are currently the mainstay of therapy for individuals living with DMD. Vamorolone, a novel GC that has recently been approved in some regions worldwide for the treatment of DMD, has also been reported to place individuals at high risk of adrenal suppression in a dose-dependent fashion, requiring health care professional awareness. Vamorolone is a mineralocorticoid receptor antagonist, which differentiates it from classic GCs, and this characteristic impacts the approach to adrenal suppression management. This contemporary perspective provides insights into the mechanisms underlying adrenal suppression due to both classic GCs and novel vamorolone therapy, followed by an overview of adrenal suppression management with a particular focus on the unique aspects of providing care for individuals treated with vamorolone. It also emphasizes the importance of educating the DMD community and health care providers about the recognition and management of adrenal suppression and outlines critical concepts for clinicians managing adrenal suppression risk, tapering GCs, and transitioning from classic GC therapy to vamorolone. The key principles of managing adrenal suppression due to classic GCs and novel vamorolone therapy highlighted in this perspective are expected to enhance clinical practice, mitigate mortality, and optimize health outcomes for individuals with DMD.
Document Type: article in journal/newspaper
File Description: application/pdf
Language: unknown
Relation: https://eprints.ncl.ac.uk/310104; https://eprints.ncl.ac.uk/fulltext.aspx?url=310104/4C2C904E-559E-4EDA-83A2-36E6BA0ADC11.pdf&pub_id=310104
Availability: https://eprints.ncl.ac.uk/310104
Rights: https://creativecommons.org/licenses/by-nc-nd/4.0/
Accession Number: edsbas.A9E9BA5E
Database: BASE