International Niemann–Pick Disease Alliance


  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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The Impact of NPC

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