Abeona Ends Enrollment For MPS IIIA Clinical Trial 

Apr 29, 2022 | Clinical Trials, News

We are disappointed to hear that Abeona Therapeutics has decided to end enrollment for their clinical trial (Study ABT-003) for MPS IIIA children aged 2-18 years with a Developmental Quotient score less than 60. 

The company has recently conducted a review of available neurocognitive data from this study, and unfortunately, improvements in participants’ neurocognitive function have not been seen. Study doctors have reiterated that they did not see a cognitive treatment effect in ABT-003 participants. Abeona has thus made the difficult decision to end enrollment for Study ABT-003. For all children enrolled in Study ABT-003, Abeona is creating an annual safety monitoring program with local physicians in the child’s country of residence.


Interested and impacted members of the Sanfilippo A community may contact Abeona at Sanfilippo@AbeonaTherapeutics.com for more information and guidance.