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VPRIV READY

LIBBY — PEDIATRIC PATIENT*

*Patient profiles for illustrative purposes only and do not depict actual patients

female patient

Libby’s parents discovered they were carriers of a type 1 Gaucher disease GBA gene mutation when they opted to do a carrier screening before her mother became pregnant with Libby.1

Libby underwent newborn screening and was diagnosed with type 1 Gaucher disease with the Asn409Ser mutation.3,4

She underwent routine monitoring every 6 months. Her doctors monitored her spleen and liver volumes, bone marrow infiltration, hemoglobin, and platelet counts.5 They also routinely took blood tests to monitor the following: glucosylsphingosine (lyso
GL-1), chitotriosidase (CHIT1), chemokine ligand 18 (CCL-18), tartrate-resistant acid phosphatase (TRAP), and angiotensin-converting enzyme (ACE).5,6

By age 5, Libby’s tests showed evidence for an enlarged spleen and abnormal platelet counts1, and her doctor recommended initiating ERT. After discussing the risks and benefits of therapy with her doctor, Libby’s parents selected VPRIV, and Libby started receiving infusions before entering elementary school.

consider
  • VPRIV has been evaluated in 24 pediatric patients (aged 4–17 years) during clinical trials (Study 044)7

CLICK ON EACH PATIENT BELOW TO READ THEIR STORY:

These are hypothetical patient profiles intended to represent patients with type 1 Gaucher disease

IMPORTANT SAFETY INFORMATION

Hypersensitivity reactions, including anaphylaxis, have occurred. The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions.

Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV.