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  1. Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

    • 21.12.23
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  2. TransportNPC: Scott Fine, CEO of Cyclo Therapeutics

    • 24.05.22
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  3. Webinar: The impacts of olipudase alfa on pediatric patients with ASMD and their families

    • 13.05.22
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  4. Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency

    • 28.03.22
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  5. Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

    • 24.03.22
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  6. Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease type C

    • 22.03.22
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  7. Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C

    • 01.11.21
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  8. Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

    • 05.02.21
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  9. A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia

    • 25.01.21
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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

    • 06.01.21
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