Current Clinical Trials

Clinical trials are where the rubber meets the road, scientifically.

It’s the crucial step of determining whether earlier research (basic and translational) is able to show the same therapeutic benefits in people.To stay current on newly emerging clinical trial listings, you can periodically search for Sanfilippo or MPS III on these sites:

The following are clinical trials currently underway or preparation stages.

If you would like more information about any of these, or about other earlier-stage research underway, contact us at

Clinical Trials: Gene Therapy

EGT-101 Gene Therapy | Type IIIA | Phase I-II | Esteve

EGT-101 is Esteve's potential first-in-class AAV-9 gene therapy that is administered via a one-time intracerebroventricular injection into the cerebrospinal fluid. EGT-101 consists of an AAV9 vector containing the human sulfamidase (SGSH) transgene and it aims to...

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Clincal Trials: Enzyme Replacement

Clincal Trials: Stem Cell Therapy

Clincal Trials: Substrate Reduction Therapy