Original Publication Date: >5 June, 2019
Publication / Source: Neurodegenerative Disease Management
Authors: Mohammed Al Jumah, Mohammad Al Muhaizea, Ahmed Al Rumayyan et al.
A multidisciplinary group of specialty experts in Duchenne muscular dystrophy treatment from the Middle East and north Africa (MENA) recently met to discuss the optimal management of this disease in the MENA region. This White Paper, published in Neurodegenerative Disease Management, presents the findings from this meeting, providing recommendations on diagnosis, treatment approaches, diseases awareness and patient education aimed to improve the standards of care for individuals with DMD.
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.