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Metabolic Disorders in Children

Metabolic Disorders in Childre

What Are Metabolic Disorders?

Metabolic disorders are conditions that affect how the body converts food into energy. They are usually caused by genetic mutations that affect enzymes or chemicals needed for metabolism. If these enzymes are missing or not working correctly, harmful substances can build up in the body, or essential substances may be missing.

Who Gets Metabolic Disorders?

Most metabolic disorders are inherited, meaning they are passed from parents to children through genes. They can affect children of all ages, but many are diagnosed in infancy or early childhood, often through newborn screening programs.

Common Types of Metabolic Disorders in Children

Here are a few major categories with examples:

1. Amino Acid Disorders

These affect how the body breaks down proteins.

  • Phenylketonuria (PKU): Buildup of phenylalanine can cause brain damage if untreated.
  • Maple Syrup Urine Disease (MSUD): Affects branched-chain amino acids, causes sweet-smelling urine and neurological damage.
  • Homocystinuria: Leads to high levels of homocysteine, affecting the eyes, bones, and brain.

2. Carbohydrate Metabolism Disorders

  • Galactosemia: Inability to process galactose (milk sugar); can cause liver damage, cataracts, and intellectual disability.
  • Glycogen Storage Diseases (GSD): Trouble storing or releasing sugar; can cause low blood sugar, enlarged liver, and growth problems.

3. Fat Metabolism Disorders

  • Medium-chain acyl-CoA dehydrogenase deficiency (MCADD): Trouble using stored fat for energy, especially during fasting.
  • Carnitine deficiency: Affects energy production in muscles and heart.

4. Organic Acid Disorders

  • Propionic acidemia, methylmalonicacidemia: Build-up of toxic organic acids; can cause vomiting, lethargy, seizures, and coma.

5. Mitochondrial Disorders

Affect the mitochondria, the energy-producing parts of the cell. Symptoms vary, including:

  • Muscle weakness
  • Developmental delays
  • Seizures
  • Hearing/vision loss

6. Peroxisomal Disorders

  • Zellweger syndrome: Affects brain, liver, and kidneys; usually severe and diagnosed in infancy.

Common Signs & Symptoms

Symptoms depend on the type of disorder but may include:

  • Poor feeding, vomiting
  • Low blood sugar (hypoglycemia)
  • Lethargy or excessive sleepiness
  • Seizures
  • Delayed growth or development
  • Muscle weakness or floppy muscles
  • Jaundice or liver enlargement
  • Unusual urine or body odor