Understanding Your Metabolic Disorder Medications

Introduction

Metabolic disorders affect how your body processes nutrients and maintains essential functions. Proper medication management is crucial for your health.

Common Medication Categories

1. Enzyme Replacement Therapies

• Purpose: Replace missing or deficient enzymes
• Administration: Usually given by infusion
• Examples: Alglucosidase alfa, Imiglucerase

2. Substrate Reduction Therapy

• Purpose: Reduces harmful substance buildup
• Administration: Often taken orally
• Examples: Miglustat, Eliglustat

3. Metabolic Modifiers

• Purpose: Help regulate metabolic pathways
• Administration: Oral medications
• Examples: Sodium phenylbutyrate, Nitisinone

Important Medication Guidelines

Storage

• Keep medications at recommended temperatures
• Protect from light and moisture
• Check expiration dates regularly

Administration

• Take medications exactly as prescribed
• Use proper timing with meals if required
• Never skip doses without consulting your doctor

Monitoring

• Regular blood tests may be needed
• Track side effects in a diary
• Schedule follow-up appointments as recommended

Warning Signs

Contact your healthcare provider if you experience:

• Unexpected side effects
• Changes in symptoms
• Problems with medication administration

Resources

• Emergency contact information
• Patient support groups
• Medication assistance programs