Effect of ayurvedic management on Duchenne Muscular Dystrophy - A case report

Abstract

Duchenne Muscular Dystrophy (DMD) is the most prevalent X-linked recessive neuromuscular disorder, affecting approximately 1 in 3,500 male infants. It arises from a deficiency in dystrophin—a protein encoded by the human genome’s largest gene that is essential for stabilizing the muscle sarcolemma. In the absence of dystrophin, the muscle membrane becomes fragile, leading to progressive fiber necrosis and profound muscular weakness, though the nervous system remains intact. While corticosteroids are the primary pharmacological option for modifying the disease course, their use is often complicated by side effects such as osteoporosis, cataracts, and significant weight gain. Clinically, this case involves a 12-year-old male presenting with a long-standing history of proximal weakness, characterized by frequent falls and difficulty navigating stairs or rising from a squatting position. Diagnostic investigations revealed a marked elevation in creatine phosphokinase (CPK) levels, and molecular analysis identified a hemizygous variant of uncertain significance (VUS) within the DMD gene. Here the child has been treated in the outpatient department of the tertiary Government Ayurveda Hospital, and medications were administered for one month, followed by inpatient management for one month by considering the concept of dhatvagni mandya (poor tissue metabolism), Ama (metabolic toxins), etc. There was a significant improvement in the clinical findings, which was assessed by the Vignos scale of lower extremity and quality of life by the Barthel index scale. Significant improvement in this short period of management substantiates the benefit of Ayurvedic medicines in the management of DMD.