Submitted by Anonymous (not verified) on 19 January 2024 - 13:49
Orphan designation: (2S,3R,4R,5S)-1-[5-(2-Fluoro-biphenyl-4-ylmethoxy)-pentyl]-2-hydroxymethyl-piperidine-3,4,5-triol Treatment of GM2 gangliosidosis, 15/02/2023 Positive
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