- Conditions
- Genetic Disease, TUBB4A-Related Leukodystrophy
- Interventions
- nL-TUBB4-001
- Drug
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- Not listed
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- San Diego, California
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- Conditions
- Genetic Disease
- Interventions
- Antisense oligonucleotide treatment (ASO)
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 4 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts