Recent Publications – Ottawa Pediatric Bone Health Group

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Recent Publications from the Ottawa Pediatric Bone Health Research Group

Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial May 27, 2026
BACKGROUND AND OBJECTIVES: Vamorolone demonstrated similar efficacy for Duchenne muscular dystrophy (DMD) compared with prednisone in a 24-week exploratory analysis and may reduce key side effects compared with classic corticosteroids. In this study, we compare the efficacy and anthropometric effect of vamorolone 6 mg/kg/d with prednisone 0.75 mg/kg/d and deflazacort 0.9 mg/kg/d in steroid-naïve boys […]
Paula R Clemens
Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD Consortium May 24, 2026
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 […]
Claire L Wood
Vertebral fractures and muscle function in glucocorticoid-treated individuals with Duchenne muscular dystrophy: a cohort study April 16, 2026
CONCLUSION: Our results suggest that ambulatory status and functional scores alone may not be reliable predictors for developing VFs and confirm the association with known risk factors, such as early initiation of glucocorticoid therapy and low BMD Z-scores, highlighting the need to guarantee a careful surveillance of possible VFs from the time of glucocorticoid initiation.
Anna Capasso
Caring for Gender Diverse Youth With Duchenne Muscular Dystrophy: A Multisite Case Series April 10, 2026
CONCLUSIONS: This international case series highlights delayed gender identity disclosure, complex hormonal decision-making, and the importance of coordinated, affirming, interdisciplinary care for TGD individuals with DMD. As survival in DMD improves, comprehensive neuromuscular care must address psychosocial well-being alongside physical health.
Carlos E Melendez Garcia
Real-world Effectiveness of Burosumab Across Age Groups: X-linked Hypophosphatemia (XLH) Disease Monitoring Program April 4, 2026
CONCLUSION: This analysis demonstrated the real-world effectiveness of burosumab for XLH, which appears evident across age groups.
Leanne M Ward
Assessing biomarkers of bone metabolism and the role of the interleukin-6 signaling pathway in patients with Duchenne muscular dystrophy March 27, 2026
Duchenne muscular dystrophy (DMD), a genetic disorder from lack of dystrophin, leads to osteoporosis and increased bone fragility. This study performed biomarker analyses to understand bone health in people living with DMD. Demographic data and total body less head (TBLH) bone mineral density (BMD) Z-scores were collected from 160 boys with DMD aged 6-11 years […]
Maitea Guridi
Expanding the Autosomal Recessive Hypophosphatemic Rickets Type I Carrier Phenotype and Adult Treatment with Burosumab March 13, 2026
No abstract
Noor Alhuda Sawalha
Key Principles and Disease-Specific Considerations to Guide Management of Bone Health and Osteoporosis Among Individuals With Neuromuscular Disorders: The Path Forward March 5, 2026
Given the intimate link between muscle function and the skeleton's ability to adapt to mechanical loads, it is no surprise that individuals with neuromuscular disorders (NMDs) are at risk for low-trauma (i.e., osteoporotic) fragility fractures. In this review, we highlight the interdependent relationship between muscle and bone strength and the need to assess individuals with […]
David R Weber
Bisphosphonate treatment for secondary osteoporosis in children, youth, and young adults: a systematic review and meta-analysis January 27, 2026
Chronic illnesses and their treatments in childhood pose significant risks of bone fragility due to osteoporosis. Bisphosphonates (BP) are commonly used for treatment, yet evidence remains limited and heterogeneous. This systematic review aims to consolidate current empirical evidence on BP efficacy and safety in children, youth and young adults aged ≤ 21 years with secondary […]
Hyunwoo Kim
Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone January 9, 2026
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, […]
Anne Marie Sbrocchi
Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial November 17, 2025
CONCLUSIONS: In this randomized clinical trial, navepegritide treatment resulted in statistically significantly higher annualized growth velocity in children with achondroplasia, with a similar safety and tolerability profile vs placebo. Moreover, navepegritide demonstrated additional potential health benefits beyond growth.
Ravi Savarirayan
Circulating protein biomarkers identified in two independent clinical trial cohorts of glucocorticoid-naive Duchenne muscular dystrophy patients November 14, 2025
Blood-accessible biomarkers offer promising insights into the pathogenesis of Duchenne muscular dystrophy (DMD) and other muscle diseases. Here, we quantified the relative abundance of 7,289 serum proteins using SomaScan proteomics in pre-treatment samples from 51 boys with DMD (aged 4 to
Fatemeh Ahmadiharchegani
Metaphyseal band fractures in pediatric patients treated with denosumab November 10, 2025
CONCLUSIONS AND RELEVANCE: Fractures through sclerotic metaphyseal bands may represent a distinct complication of denosumab therapy in children. Rebound hypercalcemia and fractures can co-occur, underscoring the need for close monitoring and management by pediatric bone specialists throughout treatment.
Rodrigo Montero-Lopez
Delayed diagnosis of X-linked hypophosphatemia in the absence of family history: a global unmet need November 4, 2025
X-linked hypophosphatemia (XLH) is a phosphate-wasting disorder mediated by increased fibroblast growth factor 23 (FGF23) activity. Typical clinical features are skeletal deformities, muscle weakness, stiffness, and impaired physical function. Using real-world data from the XLH Disease Monitoring Program (XLH-DMP) and the International XLH Registry, this study sought to determine whether the age at which XLH […]
Suma Uday
Automated bone age calculation is an independent predictor of vertebral fracture burden in Duchenne muscular dystrophy October 21, 2025
CONCLUSION: BoneXpert provided BA results that were similar to those generated by radiologists in individuals with DMD and that retained the clinically relevant association between BA delay and VF. We also demonstrated that lower BHI Z-scores in individuals with DMD were associated with spinal fragility.
Kim Phung
Jumping mechanography reference data in Canadian children and adolescents aged 6 to 20 years October 8, 2025
CONCLUSION: This study provides the largest jumping mechanography reference dataset to date, aimed at helping clinicians assess muscle function and evaluate musculoskeletal health in children and adolescents.
Oliver Li
Height, weight, and body mass index trajectories and their correlation with functional outcome assessments in boys with Duchenne muscular dystrophy August 31, 2025
AIM: To examine the factors influencing height, weight, and body mass index (BMI) z-scores, and the relationship between them and motor performance, in boys with Duchenne muscular dystrophy (DMD).
Marianela Schiava
Exploring the natural history of bone mineral density in Duchenne muscular dystrophy: a systematic literature review August 29, 2025
CONCLUSION: Understanding the natural history of BMD and disease progression relating to bone fragility will help to support clinical trial design to assess and improve bone strength in DMD. To our knowledge, this is the first SLR investigating BMD Z-score trajectories in individuals with DMD receiving GCs and highlights associations between age and GC treatment […]
Christian De Ford
Impacting the physical activity confidence of children with medical conditions or disabilities: A randomized controlled trial August 4, 2025
CONCLUSIONS: Youth who were confident were more likely to engage in physical activity. The in-person intervention increased participants' activity confidence. The limited impact of the virtual format suggests that implementing new skills with peers is critically important for enhancing activity confidence. Further research is required to evaluate whether confidence gains could be sustained beyond the […]
Hana Alazem
Switching from active vitamin D and phosphate supplementation to burosumab significantly corrects lower limb malalignment in pediatric X-linked hypophosphatemia June 13, 2025
X-linked hypophosphatemia (XLH) is a rare disorder of renal phosphate wasting and dysregulated active vitamin D metabolism, ultimately presenting as rickets and osteomalacia, among other manifestations. Lower extremity deformity (genu valgum and/or varum) is frequent in this pediatric population. Despite prompt active vitamin D and phosphate supplementation (active D/Pi), many patients require corrective surgery for […]
Leanne M Ward