Clinical Trial Update: An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy [Phase 1, Phase 2]

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Summary

Open-label, single-center study of an experimental antisense oligonucleotide for TUBB4A-related leukodystrophy, currently active not recruiting.

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Diseases / indications

• Tubb4a-related Leukodystrophy

Drugs mentioned

• Experimental antisense oligonucleotide

Snippet

Sponsor: University of California, San Diego | Conditions: Genetic Disease, TUBB4A-Related Leukodystrophy | Phase 1, Phase 2 | Status: ACTIVE_NOT_RECRUITING