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  1. Zevra Therapeutics Announces Publication of Four-Year Real-World Data from U.S. Early Access Program

    Longitudinal study includes first published real-world data on arimoclomol in adults with NPC and provides insights across a broad patient population

  2. Major Milestone Reached in Global Niemann-Pick Type C Clinical Trial

    Cyclo Therapeutics has announced that the final patient has now completed their last visit in the Phase 3 TransportNPC™ clinical study, which is evaluating the investigational therapy Trappsol® Cyclo™. This milestone represents the completion of the active treatment phase of the study and brings researchers one step closer to understanding whether this therapy could play a role in treating NPC. For individuals and families living with the condition, reaching this stage is an important moment, reflecting both progress in research and continued momentum towards potential new treatment options.

  3. Southern Nevada family fights rare 'childhood Alzheimer's' disease while pushing for a cure

    A 4-year-old Las Vegas boy is battling Niemann-Pick Type C, a fatal disease affecting just 4 children in Nevada. His family is fighting insurance barriers, traveling cross-country for care.

    Jordan and Jennifer Mitchell's home is filled with love. Their son Liam, just 4 and a half years old, is doing what kids his age do — giggling and playing. But inside his body, a race against time is underway.