International Niemann–Pick Disease Alliance

Updates

  1. INHERITED METABOLIC DISEASE AND CORONAVIRUS (COVID-19) Advice for patients / parents / guardians

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  2. Cyclo Therapeutics Announces Positive Feedback from FDA on its Pivotal Trial Design for Trappsol® Cyclo™ for Niemann-Pick Disease Type C1

    Company next plans to meet with European Medicines Agency in second quarter 2020 GAINESVILLE, FL – (Businesswire) – March 2, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of...

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  3. Mallinckrodt Announces Agreement in Principle for Global Opioid Settlement and Associated Debt Refinancing Activities

    Agreement in Principle with Court-Appointed Plaintiffs’ Executive Committee Representing the Interests of Thousands of Plaintiffs in the Opioid Multidistrict Litigation; Supported by a Broad-Based Group of 47 State and U.S. Territory Attorneys General Settlement Terms Include Structured Payments of...

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  4. Cyclo Therapeutics Announces Completion of Enrollment in its Phase I/II Trial to Evaluate Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

    GAINESVILLE, FL – (Businesswire) – February 20, 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 and Alzheimer’s Disease, today announced that it has completed...

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  5. Last Patient Last Visit in Cyclo Therapeutics’ Phase I Trial in Niemann-Pick Type C

    Data cleaning underway, top line results expected the first quarter of 2020 GAINESVILLE, FL – (Businesswire) – February 11, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease...

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  6. Cyclo Therapeutics, Inc. Poster Presentation of Clinical Trial Data on Niemann-Pick Type C Disease at the 16th Annual WORLDSymposium Now Available

    Data show that Trappsol® Cyclo™, the Company’s proprietary hydroxypropyl beta cyclodextrin drug, reduces levels of trapped cholesterol in liver tissue of Niemann-Pick Disease Type C Patients GAINESVILLE, FL – (Businesswire) – February 5, 2020 – Cyclo Therapeutics, Inc. (OTCQB:...

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  7. Sanofi : Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomyelinase deficiency

    Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomyelinase deficiency · Acid sphingomyelinase deficiency (ASMD) is a rare, progressive and potentially life-threatening disease for which no treatments are approved PARIS...

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  8. IntraBio Completes NPC Clinical Trial Enrollment

    OXFORD, UK / ACCESSWIRE / January 17, 2020, / IntraBio Inc, a late-stage biopharmaceutical company, is pleased to share that it has completed enrollment for its IB1001-201 Clinical Trial. IB1001-201 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic, and longterm,...

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  9. IntraBio Neuroprotection and Disease Modification for Niemann-Pick C Extension Phase Approved in Europe

    OXFORD, UK / ACCESSWIRE / January 15, 2020 / IntraBio Inc., a late-stage biopharmaceutical company, is pleased to share that the Extension Phase for the IB1001-201 Clinical Trial has been approved in all European countries where the trial is...

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  10. Orphazyme Announcement: US Early Access Program for NPC

    Copenhagen, Denmark, January 6, 2020 – Orphazyme A/S (ORPHA.CO), a biopharmaceutical company pioneering Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces the availability of an Early Access Program (EAP) in the United States (US) for its...

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