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Lookup NU author(s): Dr Claire WoodORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2026 The Author(s). Muscle & Nerve published by Wiley Periodicals LLC. Introduction/Aim: Optimizing Management of Endocrine Complications in Duchenne Muscular Dystrophy (OPTIMIZE DMD) is an international consortium of clinicians created to advance endocrine and bone clinical care in DMD. The aim of this study was to better understand current views and practices regarding investigation and management of growth and puberty concerns in individuals with DMD, relative to the 2018 Care Considerations and to inform updated guidance around endocrine care. Methods: A survey was created and sent to 47 OPTIMIZE DMD Consortium members and allied clinicians between September and November 2024. Areas surveyed included evaluation of growth, puberty, and arrested puberty/hypogonadism, and related management including use of vamorolone, growth hormone and testosterone. Results: Survey responses were received from 37 clinicians (79%). Most individuals were referred to endocrinology for growth and puberty concerns, with referral patterns contingent upon the endocrinology/multidisciplinary clinic model. Management discussions for growth concerns involved continued monitoring (95% of clinicians), glucocorticoid adjustment (54%) including vamorolone (35%), and growth hormone (41%). For pubertal delay, 88% of endocrinologists offered testosterone for pubertal induction, most commonly intramuscular 4-weekly testosterone injections. Most endocrinologists (92%) offered testosterone supplementation for arrested puberty/hypogonadism, some (24%) on a case-by-case basis. Delivery routes were more varied, with subcutaneous injections prescribed by 52%. Discussion: Compared with the 2018 Care Considerations, monitoring practices for growth and puberty remain largely consistent, while variability persists in management approaches. These findings provide important insights to inform future guidance and identify priorities for further education and research in endocrine management for individuals with DMD.
Author(s): Wood CL, Babalola F, Benjamin RW, Lam C, McAdam L, Nicolau S, Sbrocchi AM, Scavina M, Sorbara JC, Ahmet A, Apkon S, Bharill S, Fiscaletti M, Kinnett K, McMillan HJ, Merchant N, Nasomyont N, Molina MFO, Renthal NE, Smith P, Surampudi PN, Tamaroff JG, Tian C, Ward LM, Weber DR, Wong SC, Crane JL, Rutter MM, on behalf of the OPTIMIZE DMD Consortium
Publication type: Article
Publication status: Published
Journal: Muscle and Nerve
Year: 2026
Pages: Epub ahead of print
Online publication date: 24/05/2026
Acceptance date: 13/05/2026
Date deposited: 16/06/2026
ISSN (print): 0148-639X
ISSN (electronic): 1097-4598
Publisher: John Wiley and Sons Inc.
URL: https://doi.org/10.1002/mus.70288
DOI: 10.1002/mus.70288
Data Access Statement: The data that support the findings of this study are available from the corresponding author upon reasonable request.
PubMed id: 42178495
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