International Niemann–Pick Disease Alliance

Trial Updates

  1. CHMP recommends approval of Xenpozyme® (olipudase alfa), the first and only treatment for ASMD

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  2. Webinar: The impacts of olipudase alfa on pediatric patients with ASMD and their families

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  3. Orphazyme Announces Update on Regulatory Review of Arimoclomol in the European Union

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  4. IB1001-301: Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study

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

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  7. 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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  8. 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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  9. Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research

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

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The Impact of NPC

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