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Orphazyme phase 2 study of arimoclomol in Gaucher disease
2020-06-24 09:06:38

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.”

https://www.orphazyme.com/news-feed/2020/6/24/orphazyme-phase-2-study-of-arimoclomol-in-gaucher-disease-demonstrates-marked-improvements-in-key-clinical-markers