Nutritional Status and Therapy in DMD Patients

Study Objective This study aims to investigate the efficacy of a disease-specific, individualized dietary intervention in improving the nutritional status and functional abilities of Duchenne Muscular Dystrophy (DMD) patients aged 4-8 years residing in Ankara, Turkey.

Study Design This is a prospective, interventional study. Intervention

A 12-week individualized dietary intervention will be implemented for each participant. The intervention will focus on:

• Energy Intake: Caloric needs will be calculated based on the patient's age, weight, and activity level, taking into account the potential impact of corticosteroid therapy on energy expenditure.
• Protein Intake: Protein requirements will be met through a balanced diet, aiming to optimize muscle protein synthesis.
• Calcium and Vitamin D Intake: Adequate intake of these nutrients will be emphasized to support bone health and prevent osteoporosis.
• Fluid Intake: Fluid intake will be monitored to prevent dehydration and constipation.
• Carbohydrate Intake: A low-glycemic index diet will be recommended to manage blood glucose levels and insulin resistance, especially in patients on corticosteroid therapy.
• Sodium Intake: Sodium intake will be restricted to minimize fluid retention and hypertension.

Outcome Measures Changes in anthropometric measurements (body weight, height, BMI, skinfold measurements) Changes in functional abilities as assessed by the North Star Ambulation Assessment (NSAA) Changes in functional abilities as assessed by handgrip strenght Changes in micro and micro nutrient intake as assessed by three-day food consumption record Data Collection Data will be collected through face-to-face interviews with parents or primary caregivers and patients with DMD. After the first interview, a second interview will be conducted 12 weeks after the personalized diet has been planned.

Baseline Assessment:

• Demographic information
• Medical history
• Nutritional assessment (anthropometric measurements, dietary intake assessment)
• Functional assessment (NSAA)

Follow-up Assessments:

• Anthropometric measurements
• Functional assessment (NSAA)
• Dietary intake assessment Data Analysis Statistical analysis will be performed using appropriate statistical software. Descriptive statistics will be used to summarize the baseline characteristics of the participants. Parametric or non-parametric tests will be used to compare pre- and post-intervention outcomes, as appropriate.

Ethical Considerations The study will be conducted in accordance with the Declaration of Helsinki and approved by the local ethics committee. Informed consent will be obtained from the parents or legal guardians of the participants.

By addressing the specific nutritional needs of DMD patients, this study aims to improve their overall health and quality of life.

Inclusion criteria

Clinical diagnosis of Duchenne Muscular Dystrophy (DMD) Must be between 4 and 8 years old Able to ambulate independently Must be residing in Ankara province, Turkey Must be willing to participate and adhere to the dietary intervention, as confirmed by both the patient and their legal guardian(s) Must be currently receiving corticosteroid therapy

Exclusion criteria

Inability to read and write in Turkish Wheelchair-bound or unable to ambulate independently Significant liver or kidney dysfunction Scheduled for major surgery within 6 months of study enrollment Symptoms of dysphagia and swallowing difficulties Significant respiratory distress or respiratory insufficiency requiring mechanical ventilation.