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Posts filed in: "News"

  1. 26.01.21

    Go Make Memories, NPUK - Finalist at the EURORDIS Black Pearl Awards 2021

    We are proud to have been nominated for the #EURORDISAwards2021 (EURORDIS Black Pearl Awards) this year, in the category of Visual and Audio Media Award...

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  2. 25.01.21

    A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia

    We are pleased to share the following article "A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia” which describes the collaborative development of an innovative master protocol and primary outcome assessment to investigate N-acetyl-L-leucine as a potential treatment for three rare disorders, including Niemann-Pick disease type C (NPC)....

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  3. 20.01.21

    MNK Announcement Jan 2021

    We have received the following statement from Mallinckrodt Pharmaceuticals, stating their intention to discontinue clinical development of adrabetadex (VTS-270) for Niemann-Pick Type C1 disease (NPC), effective immediately. We understand that this will be very difficult news for many within our community; the NPUK team will be available if you would like to contact us: email info@npuk.org or telephone 0191 415 06 93.

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  4. 19.01.21

    UK Disability Survey

    The government has just announced a new UK Disability Survey and we want to hear from as many people as possible. They are particularly keen to hear from disabled people, their carers, friends and family but views from the wider public are also very welcome. The survey will remain open until 23rd April, and all views will be used to shape the delivery of the plans we set out in the Strategy with those that we receive by 13th February informing its development.

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  5. 05.01.21

    Cyclo Therapeutics Announces Positive Efficacy Data from Extension Protocol with Trappsol® Cyclo™ in Patients with Niemann-Pick Disease Type C

    Home-based intravenous infusions of Trappsol® Cyclo™ for up to one year show improvement in disease features or disease stabilization...

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