Enzyme Replacement Therapy
ERT is used to treat Lysosomal Storage Disorders (LSDs) which represent over 70 rare, inherited metabolic disorders. These are characterised by an abnormal build-up of toxic materials in the body’s cells because of enzyme deficiencies.
These disorders may affect different parts of the body, including the skeleton, brain (which may affect cognition and mobility), skin, heart, and central nervous system.
Families of affected individuals face emotional, financial, and caregiving challenges. Coping with chronic conditions requires substantial support. All NHS patients prescribed ERT now have these at home or a place of their choosing, away from a hospital setting, so that NHS hospital beds are saved for those in greater need which is where LPCH step in and support with and provide homecare.
We know how to give patients more independence
In additional to the clinical home infusion services we provide, our nurses can also help patients to become independent or semi-independent by teaching them how to do their own infusions (where appropriate).
Training patients or their loved ones, gives the patient even greater independence and flexibility.
Specialist clinical therapies at home
Our clinical teams currently treat the following Lysosomal Storage Disorders.
Pompe Disease
An autosomal recessive LSD leading to weakened hip and shoulder muscles.
Fabry Disease
An X-linked LSD affecting kidneys, heart, and skin.
Gaucher Disease
An autosomal recessive LSD causing enlarged liver, spleen, and low platelet count.
MPS Disorders
A group of autosomal recessive LSDs affecting various organs.
Learn more about our other nurse led treatments
Every day, our specialist clinical teams deliver a wide range of clinical therapies, for patients and their families.
