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Nutritional, Gastrointestinal and Endo-Metabolic Challenges in The Management of Children with Spinal Muscular Atrophy Type 1
authors: Antonio Corsello, Lorenzo Scatigno, Martina Chiara Pascuzzi, Valeria Calcaterra, Dario Dilillo, Sara Vizzuso, Gloria Pelizzo, et al.summary/abstract:
The management of patients with spinal muscular atrophy type 1 (SMA1) is constantly evolving. In just a few decades, the medical approach has switched from an exclusively palliative therapy to targeted therapy, transforming the natural history of the disease, improving survival time and quality of life, and creating new challenges and goals. Many nutritional problems, gastrointestinal disorders, and metabolic and endocrine alterations are commonly identified in patients affected by SMA1 during childhood and adolescence. For this reason, a proper pediatric multidisciplinary approach is then required in the clinical care of these patients, with a specific focus on the prevention of the most common complications. The purpose of this narrative review is to provide the clinician with a practical and usable tool for SMA1 patients’ care, through a comprehensive insight into the nutritional, gastroenterological, metabolic, and endocrine management of SMA1. Considering the possible horizons opened thanks to new therapeutic frontiers, nutritional and endo-metabolic surveillance is a crucial element to be considered for proper clinical care of these patients.
organization: University of Milan, Italy; University of Pavia, Italy; Children’s Hospital Vittore Buzzi, Milan, Italy; “Aldo Moro” University of Bari, Italy; University of Foggia, Italy
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