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

    August 03, 2026

    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

    June 17, 2026

    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

    March 12, 2026

    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.

  4. New insights into the mechanisms of "Niemann-Pick type C" in children

    December 06, 2024

    “We typically associate dementia with elderly people. However, there are also dementias that manifest in children and lead to death already by the age of 30 or even earlier, such as Niemann-Pick type C.”

    Dr. Sabina Tahirovic, neuroscientist at DZNE’s Munich site

  5. Notre Dame celebrates first FDA-approved treatment for Niemann-Pick Type C disease

    October 17, 2024

    The University of Notre Dame is celebrating a big medical breakthrough that’s been decades in the making. Within the past month, the FDA has announced the approval of new drugs to treat the rare disease Niemann-Pick Type C. You may remember, that’s the same disease that claimed the lives of three grandchildren of former Notre Dame football coach Ara Parseghian.

  6. After landmark rare disease approval, IntraBio ready to 'hit the ground running' with Aqneursa launch

    September 30, 2024

    With its first approval in hand, privately held IntraBio is embarking upon the difficult task of marketing a drug for an ultra-rare disease. Still, with months of prep work and outreach under its belt, the company is already proving it can hit the ground running. 

  7. IntraBio Announces U.S. FDA Approval of AQNEURSA for the Treatment of Niemann-Pick Disease Type C

    September 25, 2024

    NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives.

  8. Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C

    September 20, 2024

    MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease

  9. A Chesapeake family’s journey dealing with a rare disease

    June 20, 2024

    Ryan Petty, 8, was diagnosed with Niemann-Pick Type C last summer, a disease that affects only a handful of other children in Virginia, according to his family.

  10. Third Annual Rare Disease Patient Advocacy Summit

    October 31, 2023

    The third annual Rare Disease Patient Advocacy Summit was held on Friday, October 13, 2023 in the Downes Ballroom of Corbett Family Hall. Over 180 guests were in attendance and represented an array of patients and caregivers, leaders from the biotech industry, Notre Dame students and alumni, faculty and staff, and community members.

  11. Finding a Breakthrough for Tucker and the World

    October 30, 2023

    NICHD study on Niemann Pick Disease Type C has been making discoveries since 2006

  12. Schnell joins inaugural statewide rare disease advisory council

    September 29, 2023

    Santiago Schnell, William K. Warren Foundation Dean of the College of Science at the University of Notre Dame, has been appointed to a new statewide advisory council to explore new directions for assisting patients with rare diseases in Indiana.

  13. Niemann-Pick Disease Type C Market to Proliferate by 2032

    September 19, 2023

    The expected launch of various emerging therapies shall fuel the growth of the Niemann-Pick disease type C market during the forecast period, i.e., 2023–2032. The Niemann-Pick disease type C market size is expected to increase at a significant CAGR