Treatment options for Gaucher disease type 1
While there is no cure, if you have been diagnosed with Gaucher disease
type 1, treatment approaches are available to manage the disease,
including oral medicine. The goal of these treatments is to reduce or
prevent the buildup of GL-1. Treatment outcomes may vary among patients.
2 treatment approaches for Gaucher disease type 1:
SUBSTRATE REDUCTION THERAPY (SRT)
SUBSTRATE REDUCTION THERAPY (SRT)
- Oral medicine taken daily
- Reduces the amount of GL-1 that is
produced
ENZYME REPLACEMENT THERAPY (ERT)
ENZYME REPLACEMENT THERAPY (ERT)
- Intravenous (IV) infusions usually given every
2 weeks at an infusion center, a doctor’s
office, or at home as medically appropriate (Infusion
takes approximately 1-2 hours)
- Adds a modified version of the enzyme to help your
body break down GL-1
Most adult patients newly diagnosed with Gaucher disease
type 1 can begin treatment with an oral therapy, and it is possible for
most adult patients with Gaucher disease type 1 currently being treated
with an ERT (infusion) to switch to an oral therapy. Your doctor will
know the best course of treatment for you.
Cerdelga® (eliglustat) is the
ONLY first-line oral SRT. Talk to your doctor about
your treatment options
