International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. Orphazyme Statement to the NPC Community

    12 December 2020 Orphazyme Statement to the NPC Community Dear Global Community of NPC Patients, Families and Collaborators: You may have seen the news announced on 10 December that Kim Stratton has decided to resign as CEO of Orphazyme...

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  8. Cyclo Therapeutics Announces Pricing of $12.5 Million Public Offering

    Transaction Includes Uplisting to Nasdaq and Reverse Stock Split Gainesville, FL – (Businesswire) –December 9, 2020 – Cyclo Therapeutics, Inc. (“Cyclo Therapeutics” or the “Company”) (NasdaqCM:CYTH, CYTHW), a clinical stage biotechnology company developing a cyclodextrin platform for the treatment...

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  9. INPDR’s Strategic Business Plan sets out important steps on the path to progress

    The INPDR releases its ambitious Strategic Business Plan 2020 – 2022 setting targets to continue leading edge work and provide impetus for the future. The INPDR, a ground-breaking patient-focused rare disease-specific registry, has announced plans to build on progress...

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  10. Cyclo Therapeutics Announces Two Presentations at WORLDSymposium2021

    Conference Brings Together Medical Researchers and Health Professionals to Exchange Information on Lysosomal Diseases Gainesville, FL – (Businesswire) – 19 November 2020 Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company developing a cyclodextrin platform for the treatment...

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