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On #battendisease #awareness day our researchers Raffa and Nikolaj are wearing #orange & working hard to learn more… https://t.co/rDIgGfgoeO
Clinical Trial Programme
We are currently investigating the safety and efficacy of arimoclomol in Niemann-Pick type C (NPC). NPC is a rare, lysosomal storage disorder which affects around 1 in 150,000 newborns.
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