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Orphazyme completes rolling submission of New Drug Application to U.S. FDA for arimoclomol for Niemann-Pick disease Type C

Mallinckrodt Pharmaceuticals Statement: Adrabetadex for NPC-1

Please take a moment to read this important announcement from Mallinckrodt Pharmaceuticals in regard to the clinical trial of Adrabetadex for NPC-1. We recognise that this may be a surprising and disappointing development for our community as a whole. Please be assured that we are advocating for further, clearer information and will follow up with our response and next steps shortly.

Sandra Cowie, President, INPDA
Joslyn Crowe, Executive Director, NNPDF
Toni Mathieson, Chief Executive, NPUK