Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces topline results from a 6-month phase 2 dose-finding study in Gaucher disease with arimoclomol, an investigational Heat-Shock Protein (HSP) amplifier. The data show a dose-dependent effect of arimoclomol on certain disease-relevant clinical secondary endpoints, such as liver and spleen size. Furthermore, the data demonstrate sustained levels of arimoclomol in the cerebrospinal fluid (CSF), providing further evidence of arimoclomol’s ability to cross the blood-brain barrier.  

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