An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy (NCT07222371)

🧪 Interventions in This Study

• nL-TUBB4-001 (DRUG)

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (1 Sites)

• Rady Children's Hospital San Diego — San Diego, California

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

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💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Review the official NIH record below to find the coordinating site.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.