International Niemann–Pick Disease Alliance

Updates

  1. Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively

    First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies are part of Azafaros’ mission to develop treatments for the unmet need of patients with rare lysosomal storage disorders The...

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  2. AQNEURSA® (levacetylleucine) recommended for EU approval by the CHMP to treat Niemann-Pick Disease Type C

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  3. Zevra Submits Arimoclomol MAA to EMA for Niemann-Pick Disease Type C Treatment

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  4. Pfriegers Digest – 13th Issue

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  5. Pfriegers Digest – 13th Issue

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  6. Global Access Challenges for Niemann-Pick Therapies: Insights from an INPDA Community Survey

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  7. IntraBio Appoints Dr. Marc C. Patterson, US Chief Medical Officer

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  8. Update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses.

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  9. Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025

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  10. Pfriegers Digest – 12th Issue

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The Impact of NPC

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