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Bilateral Vestibulopathy: Strong Association With the ZNF91/LINC01224 Locus

Long-Term 30-Month Results of N-Acetyl-L-Leucine for Niemann-Pick Disease Type C

Results from a Global Phase 3 Trial (IB1001-303) Evaluating N-acetyl-L-leucine in Ataxia-telangiectasia