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FDA Approved Clinical Trials

FDA Approved Clinical Trials are underway to study treatments for Niemann-Pick disease. 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:

Other FDA Approved Clinical Trials

Clinical trial listings on

Learn how to participate in a clinical trial

Clinical Trial Update Procedure

The NNPDF posts new information regarding clinical trial updates as soon as it is received. Please review the following Clinical Trial Update procedure for alerting you to these updates as they are made:

The NNPDF office will post information received about trials and expanded access therapies from companies on the NNPDF website under the appropriate pharmaceutical company.

When information is received it is:

All updates from pharmaceutical companies are included in the next monthly e-newsletter with links back to the original posting on the website.

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