Enzyme Replacement Therapy
ERT for Rare Genetic Disorders
Enzyme Replacement Therapy provides patients with the enzymes their bodies cannot produce or produce in insufficient quantities. This essential treatment helps manage rare genetic disorders known as lysosomal storage diseases.
What is ERT?
Enzyme Replacement Therapy involves intravenous infusion of manufactured enzymes to replace or supplement enzymes that are missing or deficient. These enzymes are produced using recombinant DNA technology.
- Replaces deficient enzymes
- Prevents toxic buildup in cells
- Slows disease progression
Lysosomal Storage Diseases
These rare inherited conditions occur when the body lacks enzymes needed to break down certain substances. Without treatment, harmful materials accumulate in cells, causing progressive organ damage.
- Inherited genetic conditions
- Affect multiple organ systems
- Require lifelong treatment
Conditions Treated with ERT
Enzyme replacement therapy is the standard of care for several rare genetic disorders.
Gaucher Disease
Caused by deficiency of glucocerebrosidase, leading to accumulation of fatty substances in the spleen, liver, and bone marrow.
Fabry Disease
Caused by deficiency of alpha-galactosidase A, causing progressive damage to the heart, kidneys, and nervous system.
Pompe Disease
Caused by deficiency of acid alpha-glucosidase (GAA), affecting muscles including the heart and respiratory muscles.
Mucopolysaccharidosis Disorders
A group of conditions where the body cannot properly break down mucopolysaccharides, including Hunter and Hurler syndromes.
ERT Medications We Administer
Our nurses are trained in all FDA-approved enzyme replacement therapies
Imiglucerase (Cerezyme)
For Gaucher Disease
Agalsidase beta (Fabrazyme)
For Fabry Disease
Alglucosidase alfa (Lumizyme)
For Pompe Disease
Idursulfase (Elaprase)
For Hunter Syndrome
Laronidase (Aldurazyme)
For MPS I
Sebelipase alfa (Kanuma)
For LAL Deficiency
Why Home-Based ERT?
Since ERT requires regular infusions every 1-2 weeks, home treatment offers significant advantages for patients and families.
Comfort of Home
Receive treatment in your familiar environment
Flexible Scheduling
Infusions scheduled around your life
One-on-One Care
Dedicated nurse throughout your infusion
Reduced Infection Risk
Avoid exposure to clinical settings