International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. Orphazyme Submits European Marketing Authorisation Application for Arimocolmol for Treatment of Niemann-Pick disease type C

    Orphazyme Submits European Marketing Authorisation Application for Arimocolmol for Treatment of Niemann-Pick disease type C Follows U.S. Food and Drug Administration (FDA) acceptance of New Drug Application (NDA) with Priority Review in September 2020 ·        Regulatory decisions expected in 2021...

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  8. Cyclo Therapeutics Phase 3 Pivotal Program Can Begin Enrollment per US FDA

    Cyclo Therapeutics will now proceed in the US with its Phase 3 clinical trial of Trappsol® Cyclo™ for treatment of Niemann-Pick Disease Type C GAINESVILLE, FL – (Businesswire) – 21 October 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a...

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  9. IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C

    – IB1001 demonstrated statistically significant and clinically meaningful change in both the primary and secondary endpoints – Significant improvement in gait, fine motor skills, speech, cognition, overall functioning and quality of life reported – IB1001 rapidly improved both motor...

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  10. Pfrieger’s Digest – 03/2020 Edition

    “Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...

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