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Posts filed in: "Clinical Trials"

  1. 29.11.19

    #ChildrenAtTheHeart

    We are joining 140+ organisations in a call to all political parties to put children and young people at the heart of this General Election...

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  2. 19.11.19

    Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC)

    Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”

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  3. 31.10.19

    Cyclo Therapeutics Announces Its Publication of the Most Extensive Set of Case Study Reports To-date on Expanded Access Use of Hydroxypropyl Beta Cyclodextrin in Niemann-Pick Disease Type C

    Publication describes favorable safety profile and efficacy in neurologic and other disease features in individual NPC patients following intravenous administration of drug...

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  4. 22.10.19

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

    GAINESVILLE, FL – (Businesswire) – October 22, 2019 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), formerly CTD Holdings, Inc., a biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that it has completed patient enrollment in its Phase I trial to evaluate the safety and tolerability of Trappsol® Cyclo™ administered intravenously to Niemann-Pick Disease Type C (NPC) patients.

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  5. 14.10.19

    Study VTS301 – Study Resumption in France

    Study Title: A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease...

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