Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Nutritional, Gastrointestinal and Endo-Metabolic Traps in the Management of Children with Spinal Muscular Atrophy Type 1

Version 1 : Received: 4 June 2021 / Approved: 7 June 2021 / Online: 7 June 2021 (13:14:37 CEST)

How to cite: Corsello, A.; Scatigno, L.; Pascuzzi, M.C.; Calcaterra, V.; Dilillo, D.; Vizzuso, S.; Pelizzo, G.; Zoia, E.; Mandelli, A.; Govoni, A.; Bosetti, A.; Francavilla, R.; Indrio, F.; Fabiano, V.; Zuccotti, G.; Verduci, E. Nutritional, Gastrointestinal and Endo-Metabolic Traps in the Management of Children with Spinal Muscular Atrophy Type 1. Preprints 2021, 2021060184 (doi: 10.20944/preprints202106.0184.v1). Corsello, A.; Scatigno, L.; Pascuzzi, M.C.; Calcaterra, V.; Dilillo, D.; Vizzuso, S.; Pelizzo, G.; Zoia, E.; Mandelli, A.; Govoni, A.; Bosetti, A.; Francavilla, R.; Indrio, F.; Fabiano, V.; Zuccotti, G.; Verduci, E. Nutritional, Gastrointestinal and Endo-Metabolic Traps in the Management of Children with Spinal Muscular Atrophy Type 1. Preprints 2021, 2021060184 (doi: 10.20944/preprints202106.0184.v1).

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 a 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, gastrointesti-nal disorders and metabolic and endocrine alterations are commonly identified in patients af-fected by SMA1 during childhood and adolescence. For this reason, a proper pediatric multidis-ciplinary approach is then required in the clinical care of these patients, with a specific focus on the prevention of most common complications. The purpose of this narrative review is to provide the clinician with a practical and usable tool about 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, a nutritional and endo-metabolic surveillance is a crucial element to be considered for a proper clinical care of these patients.

Subject Areas

SMA; spinal muscular atrophy type 1; SMN1; nutritional management; enteral nutrition; pediatric gastroenterology; dysphagia; neurological disability; endocrine disorders; precocious pubarche

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