Major Categories

Major Categories of Inherited Metabolic Diseases

There are several hundred inherited metabolic diseases known today. Traditionally, inherited metabolic diseases were categorized as:

  1. Disorders of carbohydrate metabolism

    e.g., Galactosemia, Glycogen Storage Disease (GSD)

  2. Disorders of protein metabolism

    • Disorders of amino acid metabolism: e.g., Phenylketonuria (PKU)

    • Disorders of organic acid metabolism: e.g., Methylmalonic acidemia

    • Urea cycle disorders: e.g., Carbamoyl phosphate synthetase (CPS) deficiency

  3. Disorders of fatty acid metabolism

    e.g., Medium-chain acyl-CoA dehydrogenase (MCAD) deficiency

  4. Disorders of mitochondrial function

    e.g., Kearns–Sayre syndrome

  5. Lysosomal storage disorders

    e.g., Gaucher disease

In recent decades, many new inherited metabolic disorders have been discovered, leading to an expansion of these categories. Below are additional classes of congenital metabolic diseases along with prominent examples of each (note that this is not an exhaustive list):

  1. Disorders of porphyrin metabolism

    e.g., Acute intermittent porphyria

  2. Disorders of purine or pyrimidine metabolism

    e.g., Lesch–Nyhan syndrome

  3. Disorders of steroid metabolism

    e.g., Congenital adrenal hyperplasia (CAH)

  4. Disorders of peroxisomal function

    e.g., Zellweger syndrome