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. 2019 Jun;9(3):123-133.
doi: 10.2217/nmt-2019-0002. Epub 2019 Jun 5.

Current management of Duchenne muscular dystrophy in the Middle East: expert report

Mohammed Al Jumah  1 , Mohammad Al Muhaizea  2 , Ahmed Al Rumayyan  3 , Abdulaziz Al Saman  1 , Ali Al Shehri  2 , Edward Cupler  4 , Mohammed Jan  5 , Abubaker Al Madani  6 , Waseem Fathalla  7 , Pawan Kashyape  8 , Gururaj Kodavooru  8 , Khalid Al Thihli  9 , Laila Bastaki  10 , Andre Megarbane  11 , Cristina Skrypnyk  12 , Gholamreza Zamani  13 , Sylvie Tuffery-Giraud  14 , Andoni Urtizberea  15 , Carlos Ignacio Ortez González  16
Affiliations

Current management of Duchenne muscular dystrophy in the Middle East: expert report

Mohammed Al Jumah et al. Neurodegener Dis Manag. 2019 Jun.

Abstract

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.

Keywords: Duchenne muscular dystrophy; dystrophy; genetic disorders; multidisciplinary care; muscle degeneration; muscle weakness; treatment guidelines.

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Conflict of interest statement

Financial & competing interests disclosure

This work was supported by financial grants from PTC Therapeutics and Genpharm services. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Editorial assistance on behalf of the authors was provided by S Clissold of Content Ed Net and was funded by PTC Therapeutics and Genpharm services.

Figures

Figure 1.
Figure 1.. Multidisciplinary approach to Duchenne muscular dystrophy management.
DMD: Duchenne muscular dystrophy. Reproduced from [1] with permission of Elsevier Ltd.
Figure 2.
Figure 2.. Diagnostic pathway for Duchenne muscular dystrophy patients.
DMD: Duchenne muscular dystrophy. Adapted from [1].
Figure 3.
Figure 3.. The natural history of Duchenne muscular dystrophy as defined by change in 6-min walking test from baseline to 48 weeks from the placebo group in study 007. Presented by Spiegel et al. at the 41 Annual Meeting of the Society for Neuropediatric, Basel, Switzerland, 23–26 April 2015.
See this image and copyright information in PMC

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