Autosomal Dominant Leukodystrophy
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CLINICAL_TRIALS trial_result · 2025-05-04Clinical Trial Update: Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD) [Phase 1, Phase 2]
The document reports a personalized antisense oligonucleotide therapy being studied in one participant with LMNB1-mutant Autosomal Dominant …