About
Sanfilippo syndrome treatment
Areas of Focus
Categories
Gene Therapy, RNA & Peptide TherapyNJ, NY, PA
Clinical Trials (2)
View allTotal Trials
2
As Lead Sponsor
2
As Collaborator
0
Total Enrollment
40
By Status
RECRUITING: 1COMPLETED: 1
Clinical Trials Pipeline2 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2022
2023
2024
2025
2026
2027
2028
Today
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Recent Trials
NCT05825131Lead Sponsor
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
N/ARecruiting
30 participants
Started: Nov 10, 2024 · Completed: Nov 1, 2027
NCT05648851Lead Sponsor
A Natural History Study of Sanfilippo Syndrome Type D
N/ACompleted
10 participants
Started: Jan 22, 2023 · Completed: Nov 1, 2024
Locations (1)
Headquarters
Brooklyn, NY, United States
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