Current management of Duchenne muscular dystrophy in the Middle East: expert report.
Por:
Jumah MA, Muhaizea MA, Rumayyan AA, Saman AA, Shehri AA, Cupler E, Jan M, Madani AA, Fathalla W, Kashyape P, Kodavooru G, Thihli KA, Bastaki L, Megarbane A, Skrypnyk C, Zamani G, Tuffery-Giraud S, Urtizberea A and Ortez-Gonzalez CI
Publicada:
5 jun 2019
Ahead of Print:
5 jun 2019
Resumen:
Aim: Duchenne muscular dystrophy (DMD) is a severe and rare X-linked neuromuscular childhood disorder that results in functional decline, loss of ambulation and early death due to cardiac or respiratory failure. The objective of this paper is to address different aspects of the current management of DMD in the Middle East, north Africa (MENA) region, and to gather experts' recommendations on how to optimally diagnose and treat patients suffering from this disease. Methods: A group of experts (neuromuscular medicine, neuropediatricians and geneticists) convened to discuss the diagnosis and management of DMD in the MENA region. A list of practical statements was prepared by the chair of the meeting to guide the discussions around critical aspects relating to the current and future management of DMD. Results & conclusion: Ideally, DMD management should be a multidisciplinary approach. Nevertheless, few tertiary care hospitals in the region are currently able to provide the full spectrum of medical expertise and services needed by DMD patients. Clinical practice in the region remains heterogeneous. Specific guidelines for diagnosis and treatment are needed in the MENA region to improve outcomes. Disease awareness among the general public and the medical community is lacking. Now that mutation-specific therapies are being developed and more widely studied, general education programs regarding early signs and symptoms, a standardized referral and diagnosis pathway, patient registries and support groups will significantly improve the management of the disease.
Filiaciones:
Jumah MA:
King Fahad Medical City, Riyadh, Saudi Arabia
Muhaizea MA:
King Faisal Specialist Hospital Research Centre, Riyadh, Saudi Arabia
Rumayyan AA:
National Guard Health Affairs, King AbdulazizMedical City, Saudi Arabia
Saman AA:
King Fahad Medical City, Riyadh, Saudi Arabia
Shehri AA:
King Faisal Specialist Hospital Research Centre, Riyadh, Saudi Arabia
Cupler E:
King Faisal Specialist Hospital Research Centre, Jeddah, Saudi Arabia
Jan M:
Pedia Care Clinic, Jeddah, Saudi Arabia
Madani AA:
Rashid Hospital, Dubai, United Arab Emirates
Fathalla W:
Mafraq Hospital, Abu Dhabi, United Arab Emirates
Kashyape P:
Latifa Hospital, Dubai, United Arab Emirates
Kodavooru G:
Latifa Hospital, Dubai, United Arab Emirates
Thihli KA:
Sultan Qaboos University Hospital, Genetic Centre, Muscat, Oman
Bastaki L:
Kuwait Medical Genetic Centre, Kuwait
Megarbane A:
Université Saint-Joseph, Beirut, Lebanon
Skrypnyk C:
Center of Princess Al-Jawhara (ACMID), Arabian Gulf University, Bahrain
Zamani G:
Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran
Tuffery-Giraud S:
Laboratoire de Génétique de Maladies Rares IURC, Montpellier, France
Urtizberea A:
Hôpital Marin de Hendaye, Route de la Corniche, 64700 Hendaye, France
Ortez-Gonzalez CI:
Unidad de Patología Neuromuscular Servicio de Neurología, Hospital Sant Joan de Déu, Barcelona, Spain
Open Access
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