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Amino acid

An organic compound containing an amino group (NH₂), a carboxylic acid group (COOH), and any of various side groups, especially any of the 20 compounds that have the basic formula NH₂CHRCOOH, and that link together by peptide bonds to form proteins or that function as chemical messengers and as intermediates in metabolism.

Amino acid sequence

Peptide sequence or amino acid sequence is the order in which amino acid residues, connected by peptide bonds, lie in the chain. The sequence is generally reported from the N-terminal end containing free amino group to the C-terminal end containing free carboxyl group. Peptide sequence is often called protein sequence if it represents the primary structure of a protein.

Carbohydrate structure

In glycoproteins, in addition to the amino acid backbone or otherwise referred to as a protein, other molecules are conjugated to the protein in particular places, such as carbohydrates. Carbohydrates means sugars or chains of sugars. A single sugar is a monosaccharide, a long chain would be a polysaccharide, etc. These sugars are important for activity and are also part of the uptake mechanism for the protein within cells and tissues.

CHADD

Children and adults with attention deficit/hyperactivity disorder.

Glycoproteins

Any of a group of conjugated proteins that contain a carbohydrate as the nonprotein component.

Immunogenic

Producing an immune response.

Lysosomal

Membrane-enclosed compartment in cells, containing many hydrolytic enzymes; where large molecules and cellular components are broken down.

Lysosomal Storage Diseases (LSDs)

Are a group of which over forty disorders are currently known that result from defects in lysosomal function.

MHRA

Medicine and Healthcare products Regulatory agency.

Molecule

The smallest particle of a substance that retains the chemical and physical properties of the substance and is composed of two or more atoms; a group of like or different atoms held together by chemical forces.

MPS Society

Mucopolysaccharidosis Society.

Mucopolysaccharidosis

One of a series of inherited metabolic disorders affecting a type of complex carbohydrate called a mucopolysaccharide that is deposited in body tissues because the person lacks the specific enzyme needed to metabolize it.

NICE

UK National Institute of Clinical Excellence.

Pharmacovigilance

The pharmacological science relating to the detection, assessment, understanding and prevention of adverse effects, particularly long-term and short-term side effects, of medicines (Source: The Importance of Pharmacovigilance, WHO 2002).

Phase 1

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

Phase 2

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

Phase 3

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

Pivotal study

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

Platelet

A constituent of blood that help form clots to control bleeding.

Pre-clinical study

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

Proof of Concept study

A study intended to show that a hypothesis or concept is valid.