About
Small molecule and gene therapy treatments for lysosomal storage disorders, repurposed drugs
Areas of Focus
Categories
Small Molecule PharmaceuticalNJ, NY, PA
Clinical Trials (1)
View allTotal Trials
1
As Lead Sponsor
1
As Collaborator
0
Total Enrollment
39
By Phase
PHASE3: 1
By Status
NOT_YET_RECRUITING: 1
Clinical Trials Pipeline1 trial
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2025
2026
2027
Today
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Locations (1)
Headquarters
Paramus, NJ, United States
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