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Thinking About Experimental Therapies

Thinking About Experimental Therapies


As many families within the NNPDF membership are currently involved in clinical trials for both disease types and trials are continuing to recruit/search out more participants, this page was created to provide information for those who are thinking about participating in experimental therapies.

Follow the links below for information.


 Additional Information:

  • NNPDF Presentations on Cyclodextrin in NPC Disease – 
    • Drug Developement in Rare Diseases: the Regulatory Path to Approval
      2013 Family Conference Session – Video & Powerpoint presentation by Andrew Mulberg, M.D. of the Food & Drug Administration on drug development in rare diseases.
    • Niemann-Pick Disease Type B (ASMD): Mt. Sinai in New York
    • Niemann-Pick Disease Type C: National Institutes of Health in Bethesda, Maryland
  • In addition, please note a UK publication from Sense About Science titled, “I’ve Got Nothing to Lose.”   It is a thoughtful consideration of issues related to use of experimental therapies. Many resources listed in the booklet are in the UK, but the NNPDF Central Office can help identify similar resources in the U.S.

02/06/2017 jjb