The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.

Inclusion Criteria:

• Enzyme Replacement Therapy naive,
• confirmed diagnosis of Gaucher disease type 1 or 3,
• able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years,
• able to tolerate all study procedures,
• skeleton not fully formed as confirmed by DXA and MRI),
• and willing to receive velaglucerase alfa infusions every other week for the duration of the study.

Exclusion Criteria:

• Clinically unstable,
• taking or have taken bisphosphonates,
• Gaucher type 2,
• pregnant female,
• or deemed inappropriate for participation by the principal investigator.