Historically there was only one choice for ERT but that has changed and now we have 3 licenced treatments. The original by Myozyme by Sanofi plus a new offering and the third by Amicus Therapeutics.
The ERT contains an enzyme that naturally occurs in the body in healthy people. Those of us with Pompe lack this enzyme or the ability to make enough of it. Alglucosidase alfa helps replace this missing enzyme in people with Pompe Disease.
ERT is a standardised treatment for Late Onset Pompe Disease but it has also been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease.
Myozyme is always mixed at the time of infusion with the average person of 100Kg needing 40 bottles (20 mg per kg). With each infusion lasting 4 hours, excluding mixing times, it can take up a day every fortnight.
ERT in is administered through an intravenous infusion. Normally, once the hospital is confident that the person does not have any adverse reactions to the ERT, the infusion is organised in the persons home.
A home infusion nurse will visit fortnightly, mix the ERT to the correct dose, cannulate the person and wait until the infusion has finished.
