One-time treatment with avigbagene parvec, a gene therapy being developed by Spur Therapeutics, may improve bone health in adults with Gaucher disease. That’s according to data from a now-completed Phase 1/2 clinical trial, GALILEO-1 (NCT05324943), and its ongoing long-term extension study, GALILEO-2 (NCT06545136), showing that…
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…
