International Niemann–Pick Disease Alliance

Updates

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. 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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  8. 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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  9. 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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  10. 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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