Abstract
Objective: Pediatric residents and pediatricians play an important role in the management of Duchenne Muscular Dystrophinopathy (DMD) which is the most frequent hereditary muscle disease of childhood. Our study aims to evaluate the knowledge levels and approaches of pediatric residents and pediatricians on DMD.
Material and Methods: In this study, pediatric residents and pediatricians were asked to answer questions on the genetic, pathophysiological, clinical, and laboratory features, in addition, to follow-up and management of DMD. Data acquisition was carried out using an online questionnaire consisting of 17 questions prepared by the authors via Google forms (Google LLC, Mountain View, Ca, USA).
Results: The distribution of 197 responders was as follows: 53.8% were pediatricians, 13.7% were pediatric subspecialty fellows and 32.5% were pediatric residents with a total of 197 responders. 74.6% of the responders gave correct answers on the X-linked inheritance of DMD, 42.6% on the fact that it affected both genders, 93.3% on the fact that the disease is caused by the primary deficiency of dystrophin protein. 91.9% of the responders reported that the patients lost the ability to walk around 9-11 years of age. More than 50% of the responders did not have adequate information on the departments that could participate in the management of DMD patients.
Conclusion: This study has evaluated a wide range of physicians playing important roles in the follow-up and management of pediatric patients and has revealed a necessity for improvement in knowledge about genetic and clinical features of DMD and its management via learning.
Keywords: duchenne muscular dystrophy, neuromuscular disease, rare disease, knowledge, attitude
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