A long-term commitment

Patients and caregivers need to understand that staying on track with Aldurazyme infusions is an important part of the overall MPS I management.^1-3

• Patients may need to receive ongoing infusions of Aldurazyme, even if their symptoms improve^3,4
• If long-term treatment is stopped, GAG levels may rise again, creating a recurrence of MPS I signs and symptoms^3,4

When discussing MPS I with your patients, cover the following points^1,5:

• MPS I is a lifelong disease

MPS I patients need long-term care. It is important that we understand and address patients' concerns for better long-term care.

• Regular therapy is important

You could explain in patient-friendly terms that people with MPS I are deficient in the enzyme IDUA, which is responsible for breaking down the accumulation of GAG. When Aldurazyme is administered, it supplies the body with a source of the deficient enzyme and helps break down that accumulation of GAG. If the therapy is stopped, GAG can once again buildup, which may lead to a recurrence of certain signs and symptoms.

• Continuing therapy

It is important to continue to monitor your patients' disease progression. Due to the progressive nature of MPS I, patients may need to receive ongoing infusions of Aldurazyme, even if their symptoms have improved.

• Expectations of therapy

Patients and their families may expect quick results with Aldurazyme therapy. Consider explaining how results vary based on each patient’s own condition and response to the treatment. To maintain treatment benefit, Aldurazyme should be administered weekly as indicated.

GAG = glycosaminoglycan;
MPS I = mucopolysaccharidosis type I.