Duchenne muscular dystrophy (DMD) is an X-linked recessive degenerative neuromuscular disorder due to a deficiency of dystrophin protein. This protein is most common in skeletal and cardiac muscles, to a lesser extent in smooth muscles and the brain. With DMD, progressive damage and muscle degeneration, a delay in motor development, and respiratory cardiac disorders are progressing. Patients with DMD have an increased risk of developing osteoporosis, fractures of the tubular bones and vertebrae, and neurocognitive impairment. Vitamin D is recommended prophylactically for DMD since many studies have shown its deficiency. The purpose of this work is to consolidate the literature data on the vitamin D deficiency in DMD patients and its effects on the development of concurrent comorbid conditions of the musculoskeletal, endocrine, and nervous systems. The authors discuss data concerning the appropriate level of vitamin D throughout the life span of DMD has a positive effect on the course of the disease patients’ quality of life ends. Primary clinical outcomes of vitamin D normalization include prevention of the development of osteoporosis (especially after the start of steroid therapy), fractures of the tubular bones and vertebrae, prolonged ability to walk, more effective treatment with bisphosphonates, including a decrease in the number of complications during initial use and lower jaw necrosis, positive effect on the expression of autistic spectrum symptoms. For patients with long-term steroid therapy, metabolic and liver disorders, calcidiol could be used, allowing quick deficiency compensation instead of standard vitamin D preparations.
All-Cause Mortality and Cardiovascular Outcomes With Prophylactic Steroid Therapy in Duchenne Muscular Dystrophy
