Utilizing a Multidisciplinary Team to Improve DMD Outcomes

EP: 1.Navigating Duchenne Muscular Dystrophy: An Early-Childhood Case Example

EP: 2.The Critical Role of Dystrophin in the Pathophysiology of Duchenne Muscular Dystrophy

EP: 3.Newborn Screening and Primary Care Awareness for Timely Referral

EP: 4.Early Recognition of Duchenne Muscular Dystrophy: Key Signs Across Developmental Domains

EP: 5.Navigating the Heterogeneous Progression of Duchenne Muscular Dystrophy

EP: 6.Screening and Managing Comorbidities for Patients With Duchenne Muscular Dystrophy

EP: 7.Bridging the Gaps in Duchenne Muscular Dystrophy: Multidisciplinary Approaches for Better Outcomes

EP: 8.Navigating the Complexities of Adolescent Duchenne Muscular Dystrophy

EP: 9.Understanding the Causes of Duchenne Muscular Dystrophy

EP: 10.An Overview on The Root Causes of Duchenne Muscular Dystrophy

EP: 11.The Impact of Dystrophin Levels on Duchenne Muscular Dystrophy Progression

EP: 12.The Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy

EP: 13.The Importance of Early Intervention in Duchenne Muscular Dystrophy

EP: 14.Case 2 Overview: Patient at 16 with Duchenne Muscular Dystrophy

EP: 15.Considering New Duchenne Muscular Dystrophy Therapies: Exon Skipping and Gene Therapy

EP: 16.Expert Perspectives on The Importance of Multidisciplinary Care for Patients With Duchenne Muscular Dystrophy

EP: 17.The Future of Duchenne Muscular Dystrophy Treatment: Expert Insights on Gene Therapy

EP: 18.Employing Novel Therapies for the Treatment of DMD

EP: 19.Corticosteroid Use in Duchenne Muscular Dystrophy Management

EP: 20.The Role of Dystrophin Restoration and Gene Therapy in Cardiac and Pulmonary Function

EP: 21.Therapies to Preserve Function and Quality of Life

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EP: 22.Utilizing a Multidisciplinary Team to Improve DMD Outcomes

EP: 23.Cardiac and Pulmonary Care in DMD

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This segment introduces a 16-year-old patient representing the postambulatory phase of Duchenne muscular dystrophy (DMD), diagnosed at age 4 with a stop mutation in exon 9. The case illustrates typical disease progression: early steroid initiation, attention deficit/hyperactivity disorder diagnosis requiring methylphenidate, progressive wheelchair dependence by age 11, and emergence of cardiac arrhythmias and scoliosis in teenage years. The patient demonstrates classic complications including social withdrawal, increasing dependency for activities of daily living, and upper extremity weakness.

John Brandsema, MD, addresses the common family perception that neurology visits become unnecessary as the disease progresses, emphasizing that comprehensive interdisciplinary care remains crucial throughout all disease phases. The challenge of social isolation requires careful evaluation to distinguish between adaptive coping mechanisms (such as gaming communities that provide social interaction without physical limitations) and concerning signs of depression requiring intervention. Red flags include sleep disturbances, appetite changes, loss of interest in previously enjoyed activities, and any expressions of self-harm.

The multidisciplinary approach at specialized centers includes neurology-based teams with physical/occupational therapy, social work, genetic counseling, and nutrition support, plus collaborations with cardiology, pulmonology, orthopedics, gastroenterology, and palliative care specialists. This comprehensive care model addresses the complex medical needs that develop as patients age, including ongoing steroid management, bone health monitoring, and optimization of quality of life. The segment emphasizes that engagement with specialized care teams significantly improves outcomes for patients with DMD.