International Niemann–Pick Disease Alliance


  1. Think Again. Think NPC at the SSIEM Annual Symposium in Lyon, France

    Last week, our campaign to improve diagnosis of Niemann Pick Type C (NP-C) went on the road to Lyon, France, to take part in the SSIEM’s (Society for the Study of Inborn Errors of Metabolism) Annual Symposium. Representatives from...

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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. 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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  4. 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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  5. “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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  6. 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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  7. 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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  8. Ebola’s “Achilles heel” and link to NP-C

    Ebola’s ‘Achilles heel’ has been identified by a team of scientists, prompting hopes of new treatments to stop the virus spreading. Researchers in New York believe they have located the vital protein, which affects whether or not the virus...

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  9. Trials: A Desperate Fight to Save Kids & Change Science

    For six years, The Wall Street Journal followed a group of parents and scientists seeking a treatment for a rare and fatal genetic disease that strikes primarily children. Their collaboration accelerated development of a promising drug and, along the...

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  10. BBC News article features Think NPC campaign

    An article highlighting the INPDA’s campaign to improve diagnosis of NP-C is featured in today’s BBC News. Article can be viewed here:

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