08.01.19
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...
Read more11.12.18
Orphazyme expects to announce results of full data set for Niemann-Pick disease Type C (NPC) Phase II/III trial in Q1 2019...
Read more16.11.18
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!
Read more04.10.18
CTD announces encouraging top-line data from two ongoing clinical trials of Trappsol® Cyclo™ administered intravenously in Niemann Pick Disease Type C (NPC)
Read more03.10.18
ORPHAZYME REPORTS ENCOURAGING ARIMOCLOMOL CLINICAL TRIAL TOP-LINE DATA IN NIEMANN-PICK DISEASE TYPE C (NPC)...
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