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News

  1. 11.01.19

    CTD Announces Presentation at Upcoming Conference on Lysosomal Storage Diseases

    CTD Holdings: Annual conference offers opportunities for academic researchers and industry representatives to exchange information on novel therapeutic interventions...

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  2. 08.01.19

    Sanofi Genzyme: ASCEND and ASCEND-Peds Studies Complete Enrollment

    Sanofi Genzyme’s Global Project Team would like to announce that our pivotal registration study (ASCEND Study) for adult patients with chronic, systemic acid sphingomyelinase deficiency (ASMD) Types B and A/B (or intermediate type) and our pediatric study (ASCEND-Peds) also treating Type B and A/B ASMD pediatric patients have successfully completed enrollment for the targeted number of patients for each of these trials...

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  3. 11.12.18

    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...

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  4. 30.11.18

    Mallinckrodt Responds to Global Community Questions on VTS-270 Trial

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  5. 16.11.18

    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!

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