Tubb4a-related Leukodystrophy

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CLINICAL_TRIALS research · 2025-10-29
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]
Open-label, single-center study of an experimental antisense oligonucleotide for TUBB4A-related leukodystrophy, currently active not recruit…