Clinical Trial: MPS IIIA Disease Monitoring Program

U.S., Oct. 3 -- ClinicalTrials.gov registry received information related to the study (NCT07855562) titled 'MPS IIIA Disease Monitoring Program' on Sept. 25.

Brief Summary: The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.

Study Start Date: Nov., 2026

Study Type: OBSERVATIONAL

Condition: MPS IIIA Sanfilippo Syndrome Sanfilippo A Mucopolysaccharidosis III

Intervention: OTHER: No Intervention

No investigational/study product will be administered in this DMP.

Recruitment Status: NOT_YET_RECRUITING

Sponsor: Ultragenyx Pharmaceutical Inc

Disclaimer: Curated by HT Syndication....