Submitted by Anonymous (not verified) on 26 March 2026 - 12:59
Orphan designation: biotin Treatment of biotin-thiamine-responsive basal ganglia disease (BTBGD), 12/09/2025 Positive
Source:
Orphan designation: biotin Treatment of biotin-thiamine-responsive basal ganglia disease (BTBGD), 12/09/2025 Positive