Orphazyme Phase 2 Study of Arimoclomol In Gaucher Disease
24 June 2020
Sharing news from Orphazyme A/S, our CEO Tanya Collin-Histed writes:
“The news is a step forward for Gaucher disease patients and their families. We are excited to hear that there was evidence of arimoclomol’s ability to cross the blood-brain barrier.
Due to the success of ERT, type 3 patients are living longer however the impact of the neurological disease can be debilitating and in some cases fatal. As a community we are grateful to Orphazyme for their commitment in this area of huge unmet need.”