New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression Arimoclomol was well tolerated during the Open Label Extension trial and Early Access Program with no safety signals identified PK modeling studies showed that administration of OLPRUVA while fasting vs fed results in higher drug exposure which may allow for lower effective dosages w ...