Lysosomal storage disorders are rare inherited metabolic disorders that result from enzyme deficiencies. Enzymes in lysosomes help the body break down waste and substances it no longer needs, like worn-out cell parts. This, in turn, can disrupt normal cell function and affect many different areas of the body including the brain, heart, liver, skeleton, etc..
Although enzyme deficiencies are chronic and complex, they are often treatable with infusion therapies, usually administered every 2–3 weeks. Several replacement therapies are available, but requirements vary significantly depending on the specific disease, the age of the patient, and many other factors.
Treatment plans are complex and challenging for specialty pharmacies to facilitate. To support specialty pharmacies managing care for enzyme deficiency patients, we released an Enzyme Deficiency Care Plan. The care plan contains specific pathways of care for each disorder, making it easier for the pharmacy to track its patients’ improvements, manage their symptoms, and monitor their functional capacity. It streamlines the various factors that can influence an individual patient’s treatment journey, making it easy for pharmacists to manage care for each patient, every step of the way.
This care plan helps with:
Contact Us for more information on TherigySTM's Enzyme Deficiency Care Plan.