Pastores et al outlined therapeutic goals for a child with Gaucher Disease. Some of the goals involve growth and skeletal bone health.
Goals for growth and development:
(1) To achieve an acceptable height, ideally normal for the population.
(2) To experience the normal onset of puberty.
Goals for bone health:
(1) To improve bone mineral density, ideally being normal.
(2) To reduce or eliminate bone pain.
(3) To prevent bone crises.
(4) To prevent osteonecrosis and subchondral joint collapse.
