Core Conditions

Carnitine Uptake Defect (CUD)

Carnitine uptake defect (CUD) is an inherited condition in which the body cannot bring enough carnitine, a substance that helps the body convert energy from fats into the cells. Individuals affected by CUD are unable to break down certain fats,

Medium-Chain acyl-CoA Dehydrogenase Deficiency (MCAD)

Medium-chain acyl-CoA dehydrogenase deficiency (MCAD) is a condition in which the body is unable to break down certain fats. Individuals affected with MCAD are unable to convert some of the fats they eat into energy the body needs to function.

Very Long-Chain Acyl-CoA Dehydrogenase Deficiency (VLCAD)

Very-long-chain acyl-CoA dehydrogenase deficiency (VLCAD) is a condition in which the body is unable to break down certain fats. Individuals affected by VLCAD are unable to convert some of the fats they eat into energy the body needs to function.

Trifunctional Protein Deficiency (TFP)

Trifunctional protein deficiency (TFP) is a condition in which the body is unable to break down certain fats. Individuals affected by TFP are unable to convert some of the fats they eat into energy the body needs to function. Instead,

Argininosuccinic Aciduria (ASA)

Argininosuccinic aciduria (ASA) is a condition that causes dangerous amounts of ammonia to build up in the body. Individuals with ASA are unable to process the waste product ammonia, which is produced when the body breaks down amino acids. ASA

Citrullinemia, Type I (CIT-I)

Citrullinemia, type I (also known as classic citrullinemia or CIT) usually becomes evident in the first few days of life. Affected babies typically appear normal at birth, but as ammonia builds up they experience a progressive lack of energy (lethargy),

Maple Syrup Urine Disease (MSUD)

Maple syrup urine disease (MSUD) is a condition in which the body is unable to break down certain proteins. The condition is named for the sweet odor of the urine of untreated babies. Individuals with MSUD have trouble breaking down

Homocystinuria (HCY)

Homocystinuria (HCY) is a condition in which the body is unable to break down certain proteins. Individuals with HCY cannot process certain amino acids, small molecules that make up proteins. These amino acids build up in the body and can

Classic Phenylketonuria (PKU)

Classic phenylketonuria (PKU) is a condition in which the body is unable to break down certain building blocks of proteins, known as amino acids. Individuals with PKU cannot break down the amino acid phenylalanine. If left untreated, PKU can cause