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Here at NPUK you will regularly find us working with other like-minded organisations, such as those that support other connected rare diseases and other charities who share our main concerns and hopes. We have a track-record of successful collaborations with organisations that operate in line with our focus and commitment to improving the lives of those affected with Niemann-Pick disease and other rare diseases.

We work and connect with other organisations both nationally and internationally, enabling us to develop a stronger, more united voice, to share knowledge and to access specialist information. Through our collaborations we will be able to achieve common goals, make a greater impact and accomplish more on behalf of those we support.

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Latest news

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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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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