International Niemann–Pick Disease Alliance

Updates

  1. Cyclo Therapeutics Presents Positive Data from Clinical Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

    Data from ongoing Phase 1 extension study shows improvement in disease features or disease stabilization with home-based intravenous infusions of Trappsol® Cyclo™ Phase 1 biomarker 24S-hydroxycholesterol demonstrates clearance of excess cholesterol from the brain after intravenous infusions Interim analysis...

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  2. Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

    Orphazyme US Investor news                                                                       ...

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  3. Adrabetadex 2021: webinar featuring Dr. Porter and Dr. Berry-Kravis discussing the Adrabetadex Program

    You can find the video recording from the Ara Perseghian Medical Research Fund’s February 3, 2021 webinar below, where Dr. Elizabeth Berry-Kravis and Dr. Denny Porter presented their Expanded Access Program data, Natural History Data, and recent longitudinal data analysis...

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  4. Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

    We are pleased to share the latest update from Cyclo Therapeutics entitled “Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021”:

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  5. 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...

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  6. INPDA Spotlight on Niemann Pick Selbsthilfegruppe Deutschland e.V.

    Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

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  7. 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...

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  8. 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 Gainesville, FL – (Businesswire) – 05 January 2021 – Cyclo Therapeutics, Inc. (NasdaqCM:CYTH and CYTHW), a clinical stage biotechnology company...

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  9. Orphazyme Provides Regulatory Update on Arimoclomol for NPC

    Copenhagen, Denmark – December 27, 2020 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, announced today the U.S. Food and Drug Administration (FDA) has extended the...

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  10. Sandy Cowie, INPDA President (Christmas Message 2020)

    The INPDA Central Office is winding down for the festive break, and is set to reopen again on Monday the 4th January 2021. To mark the end of this year we are pleased to share the following message from...

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