Validation of the Pediatric Quality of Life Inventory™, Neuromuscular Module, version 3.0 in Spanish for Argentina.

Objective: To assess the psychometric properties of the Pediatric Quality of Life Inventory™ (PedsQL™ 3.0), Neuromuscular Module, version in Spanish for Argentina, for children aged 2-18 years with neuromuscular disease.

Population And Methods: Observational, analytical, prospective validation study conducted in Hospital Garrahan between March 19th, 2019 and March 9th, 2020.

Energy expenditure, body composition, and prevalence of metabolic disorders in patients with Duchenne muscular dystrophy.

Introduction: Duchenne muscular dystrophy (DMD) is a severe muscular disease characterized by progressive loss of functional muscle mass followed by changes in body composition.

Aim: To describe body composition, resting energy expenditure (REE), and metabolic disorders in DMD patients followed-up at a tertiary care center. To analyze the association with type of steroid and ambulatory status, and to compare obese DMD patients with patients with multifactorial obesity.

[Transition experience of patients with neuromuscular disease].

Neuromuscular diseases are mostly genetic disorders, with chronic and progressive course. Affected people are at high risk of developing physical and emotional disabilities. In the last decades, the advance in technology and science has increased chronic pediatric patients survival rate, thus requiring an ongoing assistance in adult hospitals, making the transition a necessity and a challenge.