News

Orphazyme expects to announce results of full data set for Niemann-Pick disease Type C (NPC) Phase II/III trial in Q1 2019

Orphazyme expects to announce results of full data set for Niemann-Pick disease Type C (NPC) Phase II/III trial in Q1 2019...

Mallinckrodt Responds to Global Community Questions on VTS-270 Trial

NPUK News: Autumn 2018

We are happy to announce that the latest issue of NPUK News (Autumn 2018) is now available. This time around it is a bumper edition featuring everything from the Annual Family Conference to BBC Children in Need, with guest articles from Shona Beveridge, Jenny Charman, and Dave Smith...and so much more!

Mallinckrodt: VTS-270 Phase 2b.3 Data Announcement

The following letter from Mallinckrodt's Sheila Talafous, Director, Advocacy Relations and Derek Naten, Senior Director, Government Relations & Advocacy refers to the VTS-270 Phase 2b.3, as well as expanded access and compassionate use programs.

CTD announces encouraging top-line data from two ongoing clinical trials of Trappsol® Cyclo™ administered intravenously in Niemann Pick Disease Type C (NPC)

CTD announces encouraging top-line data from two ongoing clinical trials of Trappsol® Cyclo™ administered intravenously in Niemann Pick Disease Type C (NPC)