Clinical Trial Update: Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy [Phase 1, Phase 2]

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Summary

A single-participant Phase 1/2 clinical trial at Massachusetts General Hospital is testing an experimental antisense oligonucleotide for Hypomyelination with at…

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Diseases / indications

• Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum

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Sponsor: Massachusetts General Hospital | Conditions: Genetic Disease | Phase 1, Phase 2 | Status: ENROLLING_BY_INVITATION