Variant Late-infantile Neuronal Ceroid Lipofuscinosis
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CLINICAL_TRIALS trial_result · 2023-03-08Clinical Trial Update: Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease [Phase 1, Phase 2]
This clinical trial update reports results from Phase 1/2 testing of a gene therapy for children with variant late-infantile neuronal ceroid…