International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. Update on Adrabetadex – Dr. Steve Romano, Mallinckrodt’s Executive Vice President and Chief Scientific Officer

    As previously mentioned during Mallinckrodt’s  webinar on July 16th 2020, Dr. Steve Romano, Mallinckrodt’s Executive Vice President and Chief Scientific Officer,  has prepared the following video for the NP-C community which provides an update on Adrabetadex: You can learn more about...

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  5. Cyclo Therapeutics Closes $2.8 Million Private Placement Led by Novit LP with $1.0 Million

    GAINESVILLE, FL – (BusinessWire) – August 27, 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 it...

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  6. INPDA Webinar: Mallinckrodt Pharmaceuticals – Adrabetadex for NPC-1

    The International Niemann-Pick Disease Alliance will host a webinar on Sunday 26th July, at 6pm BST /7pm CET / 1pm ET. The webinar will provide an opportunity to hear from Mallinckrodt Pharmaceuticals regarding their recent announcement, which has raised...

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  7. Orphazyme completes rolling submission of New Drug Application to U.S. FDA for arimoclomol for Niemann-Pick disease Type C

    Orphazyme A/S Investor News                                                                       ...

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  8. Mallinckrodt Pharmaceuticals Statement: Adrabetadex for NPC-1

    Please take a moment to read this important announcement from Mallinckrodt Pharmaceuticals in regard to the clinical trial of Adrabetadex for NPC-1. We recognise that this may be a surprising and disappointing development for our community as a whole. ...

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  9. Pfrieger’s Digest – 02/2020 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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  10. Cyclo Therapeutics’ Clinical Data Webinar Recording Available on Company Website

    GAINESVILLE, FL – (BusinessWire) – May 26, 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, issued a press release...

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