International Niemann–Pick Disease Alliance

Trial Updates

  1. Cyclo Therapeutics Announces Formation of Global Steering Committee Comprised of Leading Experts to Advise on the Global Phase 3 Clinical Development Program for Trappsol® Cyclo™ in Niemann-Pick Disease Type C

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  2. EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD

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  3. Orphazyme announces publication of results from its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C in the Journal of Inherited Metabolic Disease

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  4. IntraBio Reports Statistically Significant and Clinically Meaningful Improvements in the Use of IB1001 For Treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease)

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  5. Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research

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  6. Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C

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  7. Orphazyme: NPC Community Notification

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  8. Cyclo Therapeutics Announces Design of Pivotal Phase 3 Study Evaluating Trappsol® Cyclo™ in Niemann-Pick Type C1

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  9. Cyclo Therapeutics Presents Positive Data from Clinical Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

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  10. Cyclo Therapeutics Announces Positive Efficacy Data from Extension Protocol with Trappsol® Cyclo™ in Patients with Niemann-Pick Disease Type C

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