Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum

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CLINICAL_TRIALS research · 2026-03-02
Clinical Trial Update: Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy [Phase 1, Phase 2]
A single-participant Phase 1/2 clinical trial at Massachusetts General Hospital is testing an experimental antisense oligonucleotide for Hyp…