FDA Approved Clinical Trials
Clinical trials are underway to study treatments for NPC. Follow the links below for information.
ASMD Clinical Trial
There are no current clinical trials for ASMD in the U.S. at this time. For information on Xenpozyme, approved for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, visit XENPOZYME® (olipudase alfa-rpcp)
NPC Clinical Trials
Active Not Recruiting:
- Zevra Therapeutics (Formerly KemPharm)
Completed or Discontinued:
Donation of Biological Samples to Assist with Research
Many families ask what they can do to help move research forward more quickly. One, of course, is to raise funds – research is expensive and every little bit helps! Another important way to help, however, and one that only the patients and families can do, is to donate biological samples for use in the research studies. This might include blood or skin cell samples taken at the time of diagnosis or other medical procedures, or blood, skin cell and tissue samples taken at the time of a surgery or at time of death.
The National Niemann-Pick disease Foundation has worked with several programs and organizations where donations will be accepted, depending upon which type of NPD affects the donor. Please select one of the following links to learn more about how to arrange a donation and review the documents that will need to be completed to allow for sample collection.
Thank you for considering this most generous of donations!
Niemann-Pick Disease, Types A and B/Acid Sphingomyelinase Deficiency
Niemann-Pick Disease, Type C
Stem Cell Information from the International Society for Stem Cell Research
There has been much recent discussion among members of the NNPDF about stem cell therapies for lysosomal storage disease. While this should not be seen as an endorsement of this approach to disease management at this time, it is important that we provide our members with unbiased information about developing technologies. The following link provides access to both basic and more in-depth information about this field of investigation: