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.
- 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.
- Clinically unstable,
- taking or have taken bisphosphonates,
- Gaucher type 2,
- pregnant female,
- or deemed inappropriate for participation by the principal investigator.
Raphael Schiffmann, M.D.,M.H.Sc.
Baylor Research Institute/Institute of Metabolic Disease