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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

 

Introduction

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.

 

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 cure or effective treatment commercially available to Sanfilippo patients, recent years have seen a major break-through progression from pre-clinical research (animal testing) to clinical trials on human patients. There are currently many research programs around the world working on MPSIII with several in clinical trial stage. In addition, numerous behavioural studies have been undertaken to better understand the disease, its cause, onset and progression path.

 

Whilst timeframes for taking a therapeutical product from bench-to-bed (from the research bench to the bedside of the patient) can be significant, there has been an acceleration of these timeframes for Sanfilippo patients, largely due to the tireless efforts of patient families and patient groups.

 

 

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 groups underway across the world please contact us if you would like any further information.