Evidence map›Paper›PMID 42857968›Full record

ReviewJournal of neuromuscular diseases2026

Managing growth and puberty concerns in individuals with Duchenne muscular dystrophy: Updated guidance and future directions.

Meilan M Rutter, Janet L Crane, Funmbi Babalola, Robert W Benjamin, Carol Lam, Laura McAdam, Stefan Nicolau, Anne Marie Sbrocchi, Mena Scavina, Julia Sorbara and 31 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

41 authors.

Meilan M RutterDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.ORCID 0000-0003-4142-9527
Janet L CraneDepartment of Pediatrics, Division of Endocrinology, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID 0000-0001-7713-0936
Funmbi BabalolaDepartment of Pediatrics, Division of Endocrine, Children's Hospital, London Health Sciences Centre, University of Western Ontario, London, ON, Canada.ORCID 0000-0002-4460-4514
Robert W BenjaminDivision of Pediatric Endocrinology, Duke University Medical Center, Durham, NC, USA.
Carol LamDivision of Endocrinology, The Hospital for Sick Children, Toronto, ON, Canada.
Laura McAdamDepartment of Pediatrics, Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Toronto, ON, Canada.
Stefan NicolauCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.ORCID 0000-0001-5631-0559
Anne Marie SbrocchiDivision of Pediatric Endocrinology and Metabolism, Montreal Children's Hospital, McGill University Health Center, Montreal, QC, Canada.ORCID 0009-0005-3682-8293
Mena ScavinaParent Project Muscular Dystrophy (PPMD), Washington, DC, USA.
Julia SorbaraDivision of Endocrinology, The Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0001-6324-6727
Alexandra AhmetDepartment of Pediatrics, Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Susan ApkonDepartment of Physical Medicine and Rehabilitation, Children's Hospital Colorado and Anschutz University of Colorado School of Medicine, Aurora, CO, USA.
Chiara ArpaiaCentro Clinico NeMO, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.ORCID 0000-0001-6193-6249
Tommaso AversaDepartment of Human Pathology of Adulthood and Childhood, Pediatric Unit, "G. Martino" University Hospital, University of Messina, Messina, Italy.ORCID 0000-0002-1754-6822
Shipra BansalDepartment of Pediatrics, Division of Endocrinology, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
Luca BelloDepartment of Neurosciences, DNS, University of Padova, Padova, Italy.ORCID 0000-0002-3075-6525
Diana X Bharucha-GoebelCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.ORCID 0000-0002-7437-0982
Sabrina CorbettaBone Metabolic Diseases and Diabetes Unit, Istituto Auxologico Italiano IRCCS, Milan, Italy.ORCID 0000-0001-8140-3175
Natascia Di IorgiDepartment of Pediatrics, Pediatric Endocrinology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Ryan FarrellDivision of Pediatric Endocrinology, University Hospitals Cleveland Medical Center, Rainbow Babies and Children's Hospital, Cleveland, OH, USA.ORCID 0009-0005-2497-5253
Alberto FerlinDepartment of Medicine, University of Padova, Padova, Italy.ORCID 0000-0001-5817-8141
Danilo FintiniEndocrinology and Diabetology Unit, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Melissa FiscalettiDivision of Pediatric Endocrinology, Department of Pediatrics, Sainte-Justine University Hospital, Université de Montréal, Montréal, QC, Canada.ORCID 0000-0003-2092-0691
Rana HallounThe Ottawa Pediatric Rare Bone Disease Research Group, Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada.
Maria-Elena LautatzisDepartment of Pediatrics and Child Health, Children's Hospital of Manitoba, University of Manitoba, Winnipeg, MB, Canada.ORCID 0009-0000-2415-8062
Hugh J McMillanDepartment of Pediatrics, Division of Neurology, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.ORCID 0000-0001-8927-2018
Nadia MerchantDepartment of Pediatrics, Division of Pediatric Endocrinology, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Patrick MoeschenParent Project Muscular Dystrophy (PPMD), Washington, DC, USA.
Nat NasomyontDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Maria Fernanda Ochoa MolinaDepartment of Pediatrics, Pediatric Endocrinology Unit, Clínica Alemana de Santiago, Santiago, Chile.
Chiara PanicucciCenter of Translational and Experimental Myology, IRCCS Giannina Gaslini Institute, Genoa, Italy.
Nora E RenthalDepartment of Pediatrics, Division of Endocrinology, Harvard Medical School, Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0002-8186-4462
Luisa De SanctisDepartment of Pediatric Endocrinology, Regina Margherita Children's Hospital, Turin, Italy.
Jaclyn TamaroffDepartment of Pediatrics, Division of Endocrinology and Diabetes, Vanderbilt University Medical Center, Nashville, TN, USA.
Cuixia TianDivision of Neurology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Aravindhan VeerapandiyanDepartment of Pediatrics, Division of Neurology, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
Leanne M WardDepartment of Pediatrics, Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.ORCID 0000-0003-1557-9185
David R WeberDivision of Pediatric Endocrinology and Diabetes, The Perelman School of Medicine at the University of Pennsylvania, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID 0000-0001-6895-2372
Sze Choong WongBone, Endocrine, Nutrition Research Group in Glasgow, Human Nutrition, University of Glasgow, Glasgow, UK.ORCID 0000-0003-2845-6555
Ilaria ZitoParent Project APS, Rome, Italy.
Claire L WoodDepartment of Paediatric Endocrinology, Royal Victoria Infirmary, Great North Children's Hospital, Newcastle upon Tyne, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Long-term glucocorticoid therapy is standard of care for individuals with Duchenne muscular dystrophy (DMD) but often results in impaired growth and delayed or incomplete puberty, impacting their physical and psychosocial health. Although DMD endocrine care has evolved, leading to inclusion in the 2018 Care Considerations, these issues remain prevalent and concerning, and gaps in care persist. The Growth and Puberty Working Group of the OPTIMIZE DMD Consortium, an international multidisciplinary group including endocrinology and neuromuscular specialists with DMD expertise, conducted a literature review and surveyed clinicians to inform updated clinical guidance on growth and puberty. This report is based on evidence where available, plus expert consensus, and thereby provides revised guidance on the investigation and management of growth and pubertal delay, as well as hypogonadism beyond the pubertal years, among individuals with DMD. It also addresses new topics subsequent to the 2018 Care Considerations, including: 1) the dissociative glucocorticoid vamorolone and its impact on growth, and 2) gonadal function in older adolescents and young adults. With the current lack of long-term data on growth and puberty outcomes, prevailing themes underscore the importance of person- and family-centered care with informed shared decision-making aligned with individualized goals plus interdisciplinary team communication. This report also highlights specific areas in these domains in need of research. Through ongoing collaborations within the OPTIMIZE DMD Consortium, growth and puberty care continues to evolve and will inform and advance research to improve the health and quality of life of individuals with DMD.

Indexed as

boneDuchenne muscular dystrophyendocrinefertilitygrowthhypogonadismpubertysteroidtestosteronevamorolone

Identifiers

PMID42857968
PMCPMC13654019

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.