International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. Pfrieger’s Digest – 3rd Issue

    “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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  8. 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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  9. 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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  10. Cyclo Therapeutics Inc. Announces Additional Efficacy Data from its Ongoing Phase I/II Trial using Trappsol® CycloTM Intravenously to Treat Patients with Niemann-Pick Disease Type C1 (NPC1)

    GAINESVILLE, FL – (BusinessWire) – September 8, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that 3...

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