Rare Diseases & Orphan Drugs

Rare nutritional and metabolic diseases

Metabolism is the chemical process which our body uses to transform the food we eat into the form energy or fuel that keeps us alive and energetic too. When the metabolism processes fails a metabolic disorder causes and results in the body to have either too high or too little of the essential materials needed to stay healthy. Our body is very sensitive to mistakes or errors in metabolism. The body must have amino acids and many types of proteins to perform all its metabolic functions.  Calcium, potassium, and sodium ions are utilized by our brain to generate electrical impulses and to send signals, and also lipids and fatty acids to maintain a healthy nervous system.