International Niemann–Pick Disease Alliance


  1. Vtesse Announces Preliminary Data from On-going Phase 1 Study of VTS-270 for Treatment of Niemann-Pick Disease Type C (NP-C)

    Vtesse, Inc. have announced the preliminary results of the open-label Phase 1 clinical trial of VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for the treatment of Niemann-Pick Disease Type C (NPC) conducted by researchers at the National Institutes of Health (NIH)...

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  2. Calporta Therapeutics is developing small molecule agonists of TRPML1 for Niemann-Pick C

    Calporta Therapeutics is developing small molecule agonists of TRPML1 for the treatment of Niemann-Pick C Disease (NPC) and other lysosomal storage diseases. Calporta’s approach is based on research from the lab of Haoxing Xu, Ph.D., at the University of...

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  3. Vtesse, Inc. Expands Scientific Advisory Board

    Vtesse, Inc. Expands Scientific Advisory Board, Fills Key Patient Advocacy Position to Prepare for Further Clinical Development of VTS-270 in Niemann-Pick Disease Type C (NPC) Gaithersburg, MD, June 15, 2015 – Vtesse, Inc., a rare disease company focused on...

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  4. Genzyme trial for NP-B – first paediatric patient dosed with investigational ERT

    Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients: First patient dosed with investigational enzyme replacement therapy designed to treat the nonneurological manifestations of ASMD which characterize Niemann-Pick disease type B. Monday, June 15, 2015...

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  5. IMAGINE short film released

    “IMAGINE” follows the story of Millie, a little girl who dreams of being a dancer, yet suffers from Niemann Pick Type C; an extremely rare, incurable disease which causes neurological decline and dementia. To watch the film, visit 17...

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  6. “Imagine” short film to be premiered this weekend 13th July

    IMAGINE follows Millie, a little girl suffering from NP-C. Imagine has  been developed in collaboration with the Hope for Hollie Foundation, NPUK and the INPDA, to raise awareness of the disease. We can’t wait to share it!

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  7. Vtesse webinar on proposed clinical trial for VTS-270 – transcript available

    The transcript from Vtesse’s webinar, held on April 16th 2015, is available at the following link:!patient-webinars/c1nf6

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  8. Orphazyme’s Prospective Non-therapeutic Study in Patients Diagnosed With NP-C

    Orphazyme’s ‘Observational’ study for NP-C has been registered and made available on The trial identifier is NCT02435030. Confirmed sites and contact details are provided. You can find further information on Orphazyme’s website  

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  9. NP-UK trustee provides a summary of NP-C trials

    INPDA member Niemann-Pick UK provides a new post from Will Evans (father of Sam, NP-C, and trustee of NPUK), giving his perspective of the planned clinical trials in NP-C: New Post! Clinical Trials in NP-C – My Perspective, by...

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  10. FDA Grants Breakthrough Therapy Designation for Genzyme’s Olipudase Alfa

    Genzyme, a Sanofi company, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa. This enzyme replacement therapy is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase...

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