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Background Vitamin D deficiency is common in Duchenne muscular dystrophy (DMD) and contributes to impaired skeletal health. Evidence describing early real-world responses to supplementation during the ambulatory phase remains limited. Methods Prospective case series including five ambulatory boys with genetically confirmed DMD receiving individualized oral cholecalciferol (vitamin D 3 ) for 6 months. Results Serum 25(OH)D increased in four of five patients, bone mineral density Z-scores improved descriptively in all patients, no new fractures occurred, and treatment was well tolerated. Conclusions Individualized correction of vitamin D deficiency appears feasible and safe in ambulatory children with DMD. These findings support routine screening and individualized correction of vitamin D deficiency as part of multidisciplinary care in ambulatory children with DMD, while highlighting the need for larger controlled studies evaluating long-term skeletal outcomes.
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Cacciaguerra et al. (2026) studied this question.
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