Glossary of terms

A drug technology platform acquired as part of the NRP acquisition.

Small molecule drugs called pharmacological chaperones are used to selectively bind to a target protein and increase its stability. The binding of the chaperone molecule helps the protein fold into its correct three-dimensional shape. This allows the protein to be trafficked from the endoplasmic reticulum to the appropriate location in the cell, thereby increasing protein activity and cellular function and reducing stress on cells. This approach was developed to address human genetic diseases resulting from misfolded proteins.

A medical treatment replacing an enzyme in patients whom that particular enzyme is deficient or absent. Usually this is done by giving the patient an intravenous (IV) infusion containing the enzyme.

US Food and Drug Administration

5-ASA (5-aminosalicylic acid) also known as Mesalazine or Mesalamine, is an anti-inflammatory drug used to treat inflammation of the digestive tract ulcerative colitis and mild to moderate Crohn's disease. Mesalazine is a bowel-specific aminosalicylate drug that acts locally in the gut and has its predominant actions there, thereby having few systemic side-effects.

Oral therapies usually conducted in healthy human volunteers to determine if a drug candidate is safe for more extensive testing.

Clinical trials conducted in patients with relevant disease to assess the safety and efficacy of the drug candidate.

Clinical trials conducted in the target patient population to comprehensively assess the safety and efficacy of the drug candidate.

A major clinical trial that has a significant impact on the labeling (approved usage) of a drug.

Studies of compounds conducted in the laboratory, in isolated tissues, or in living animals.

A disease is symptomatic when it is at a stage when the patient is experiencing symptoms. It is generally used in counter distinction of asymptomatic (when the disease is inapparent). Symptomatic treatment is the practice of treating a patient's symptom, rather than the disease or injury itself.