Inborn Errors of Metabolism (IEMs) are a large and varied group of rare inherited disorders that cause a disruption in the body’s metabolism. They are most commonly caused by a gene defect inherited from both parents, causing a deficiency in one of the body’s many enzymes.

This results in the accumulation of substances which are toxic, or in the inability to make essential compounds. IEM’s cause a wide variety of very rare illnesses, many of which have no licensed medicines available to treat them. At Veriton Pharma we are passionate about developing specialised medicinal products for these groups of patients.

IEM is a general term that can be used to describe a wide range of diseases and conditions.

One common factor between all the different IEM diseases and illnesses is that there is a genetic component, or malfunction, which results in the patient missing or having a low level of an enzyme or co-factor in a metabolic pathway.

This means that the patient may have difficulties in processing, transporting, storing or breaking down components of foodstuffs and or subsequent by-products. Depending on the missing enzyme or co-factor this could lead to a failure to release or store nutritional resources or, at a later stage in the metabolic pathway, a build-up of toxic waste materials in the body that cannot be metabolised or excreted.

Diseases and conditions linked to Inborn Errors of Metabolism are sometimes identified soon after birth.  Treatment is usually initiated in a specialist care setting, and can take the form of a patient being given regular replacement doses of a substrate or component in a metabolic pathway.

Veriton Pharma supplies a range of products used in different diseases and conditions and this range is increasing. Healthcare professionals who wish to find out more about our range of products should contact us on +44 (0)1932 690325 or send us a message using the contact us form.

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