Colchicine, an approved anti-inflammatory medication for treating gout, significantly reduced fat accumulation and signs of cellular damage in a cell model of Gaucher disease, according to a new study. The lab findings suggest that the oral treatment — which was identified for potential use in Gaucher with the aid…
VPRIV (velaglucerase alfa) may help improve bone marrow health in adults with Gaucher disease type 1, but its benefit for bone strength appears to be modest and may vary from patient to patient, a small Phase 4 clinical study found. The study, “Improvement of Bone Mineral Density…
Long-term treatment with Cerezyme (imiglucerase), or its discontinued predecessor Ceredase (alglucerase), led to rapid and progressive improvements in blood counts, growth, and bone health, as well as reductions in liver and spleen size, in children with Gaucher disease (GD) — including among youngsters with a more severe disease…
