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  1. 31.10.16

    Orphazyme: Update #11 on the AIDNPC Clinical Programme

    At the latest end-of-the-month AIDNPC telephone conference hosted by the sponsor, Orphazyme ApS, the following update on the recruitment into the ‘-001’ and ‘-002’ Studies was presented. Over the last month, no more patients have enrolled in neither of the ‘-001’ and ‘-002’ Studies, leaving the total at 35 patients enrolled to date at 12 European sites...

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  2. 28.10.16

    "No more tiredness - I look forward to it!" Rani Voncxz

    The days have been tiring recently for the family from Tessenderlo, but they endured the biweekly trips to Amsterdam for the tests with a smile! Not exactly surprising when you learn that this year marks sixteen years of searching…and that finally they have heard that treatment may be possible.,,

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  3. 28.10.16

    The Quinn Madeline Foundation Establishes First-Ever Niemann-Pick Diagnostic Carrier Screening Programme

    The Quinn Madeleine Foundation has partnered with Baby Genes Inc. to provide diagnostic carrier screening at no charge to hundreds of people impacted by Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick Disease Types A & B, a severe lysosomal storage disorder. Type A (NPA) is always fatal by toddlerhood...

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  4. 27.10.16

    Perlara & Novartis: Join Fight Against Niemann-Pick Disease

    Perlara and Novartis join the fight against Niemann-Pick Disease: Perlara was founded in February 2014 and is the brainchild of molecular biologist Ethan Perlstein. Perlara is a biotech startup based in the USA, whose mission is to discover personalized cures for diseases rare and common...

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  5. 20.10.16

    Star Online: Carers Star

    Star Online is an innovative system for supporting and measuring change when working with different client groups. It is well researched, widely used and recommended and from its initial development for working with homeless people, it now covers many different client groups. At NPUK we decided to introduce the Star...

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