Submitted by Anonymous (not verified) on 13 September 2024 - 16:46
Orphan designation: N-[(1R)-1-[(S)-(2-Chloro-3-fluorophenyl)hydroxymethyl]butyl]-7-fluoro-2,3-dihydro-2-oxo-1H-indole-4-carboxamide Treatment of congenital alpha-1 antitrypsin deficiency, 25/07/2024 Positive
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