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The coronavirus disease 2019 (COVID-19) pandemic has caused overwhelming challenges in healthcare worldwide. During such an outbreak, some needs of high-risk groups who require regular follow-ups and long-term management are not met. The vulnerable populations include patients with Duchenne muscular dystrophy (DMD). Duchenne muscular dystrophy is characterized by respiratory complications caused by muscle weakness. Hence, patients with this condition are at high risk of severe diseases including COVID-19. To standardize care and provide optimal treatment to DMD patients in Saudi Arabia during the COVID-19 pandemic, a panel of experts including neurologists and pediatricians consolidated recommendations for healthcare professionals and caregivers. During this pandemic, substituting unnecessary clinic visits with virtual clinic services was highly recommended, if possible, without compromising clinical outcomes. Duchenne muscular dystrophy patients with respiratory complications should be closely monitored, and those with cardiovascular complications must continue taking angiotensin-converting enzyme inhibitors or angiotensin receptor blockers. Moreover, individualized home-based rehabilitation management was preferred. Glucocorticoid and new gene correction therapies should be continued. However, new gene correction therapy must be post-poned in newly diagnosed patients. A multidisciplinary decision was required before the initiation of hydroxychloroquine based on the COVID-19 treatment protocol. COVID-19 has caused challenges and transformed access to health care. However, these limitations have provided opportunities for the health care system to adapt. Further, telemedicine has become a reliable platform for follow-up appointments that should be conducted by a multidisciplinary team including physicians, dieticians, and physical therapists.
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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7927788 | PMC |
http://dx.doi.org/10.3389/fped.2021.629549 | DOI Listing |
Neuro Endocrinol Lett
September 2025
Department of Pediatric Neurology, Cukurova University Faculty of Medicine, Adana, Turkey.
Objective: It is important to raise awareness of the nutritional problems that can be overlooked during the follow-up visits with children who suffer from neuromuscular diseases, as these dietary differences may lead to additional neurological and systemic problems and impair the quality of life of the patient. The aim of this study was to evaluate the nutritional status of children with neuromuscular disorders and to prevent possible complications by recognizing possible nutritional problems in advance.
Methods: Patients who applied to the outpatient clinic at Cukurova University, Faculty of Medicine, Department of Pediatric Neurology beginning in April 2022 with a neuromuscular disorder diagnosis were followed up with and were included in the study.
Health Expect
October 2025
JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong, China.
Objective: This study examined the content validity of EQ-5D for Chinese patients with DMD. Specifically, it investigated: (1) the content validity of EQ-5D-5L in adult DMD patients and (2) the content validity of EQ-5D-Y-3L (Y-3L) in DMD patients aged 8-15 years.
Method: This qualitative study used semi-structured interviews and conducted one-on-one and online, with two groups of individuals with DMD to examine the content validity of the EQ-5D-5L and Y-3L.
Mol Ther Methods Clin Dev
June 2025
Eli and Edyth Broad Stem Cell Center, University of California, Los Angeles, Los Angeles, CA, USA.
Expert Rev Cardiovasc Ther
September 2025
Department of Cardiac Surgery, Thomas Jefferson University, Philadelphia, PA, USA.
Background: Heart transplant (HTx) in dystrophy patients has been shown to have a similar survival to cardiomyopathy from other causes, but postoperative rehabilitation remains an issue. This study aimed to review and analyze the reports in the literature to determine whether pre- and post-transplant functional status along with wheelchair dependence in dystrophy patients can influence post-HTx outcomes.
Research Design And Methods: Relevant databases were queried for all case reports and case series regarding HTx in patients with dystrophy-associated cardiomyopathy published in the literature.