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Current Research Programs

Since it was first described by American physician Sylvester Sanfilippo in 1963, much research has gone into understanding the disease and investigating approaches for effective therapeutic treatments. 

   Gene Therapy   |   Stem Cell   |   Enzyme Replacement  |   Substrate Reduction



In this section, we share information on current research programs with a focus on those in, or about to enter, clinical trials stage . For a description on the different types of potential therapies for Sanfillippo, please see our Therapy Avenues section.


Although there are currently no cures or effective treatments commercially available to Sanfilippo patients, recent years have seen a major progress with research moving from pre-clinical (animal testing) to clinical trials on human patients. In addition, numerous studies have been undertaken to better understand the disease, its cause, onset and progression path.

Research Programs in Clinical Trial Stage

You can read about research programs that are either in or about to enter human clinical trial stage by clicking on the Gene Therapy, Stem Cell, Enzyme Replacement Therapy and Substrate Reduction menus (above and on left).


Click here to view our global Research Program Map (a one-page summary of research programs in or about to enter clinical trial.)


Please note, there are many other earlier stage research studies underway across the world please contact us if you would like any further information.