International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. 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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  8. 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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  9. IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C

    IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C Positive results reported from IntraBio’s multinational clinical trial with IB1001 for treatment of Niemann-Pick disease Type C (NPC) Statistically significant and clinically...

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  10. Orphazyme announces U.S. FDA acceptance and Priority Review of New Drug Application for arimoclomol for Niemann-Pick disease Type C

     If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) FDA has set target PDUFA action date of March 17, 2021 Copenhagen, Denmark, September 16, 2020 – Orphazyme A/S (ORPHA.CO), a...

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