Arimoclomol treatment. On May 15, 2022, KemPharm, Inc., a specialty pharmaceutical company focused on rare central nervous system diseases like NPC, announced its acquisition from Orphazyme of Arimoclomol. This action supports the confidence of NPC clinicians in the benefit of Arimoclomol. “We believe the efficacy signal for Arimoclomol in NPC is convincing and that there …
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Research News
New biotech Mandos to take over Adrabetadex (Cyclodextrin) drug trial
Adrabetadex (Cyclodextrin) Clinical Trial. As many of you may know, much to the surprise of the NPC community, Mallinckrodt in January 2021 abandoned the Adrabetadex clinical trials; which Andrew Marella has been a part of for over 7 years. Much of the NPC community’s efforts since that announcement had been focused on what would come …