@drugsincontext.bsky.socialđź’Š Biochemical and clinical changes during enzymatic replacement therapy in hypophosphatasia: three real-world cases in Spain
Marbán Calzón et al.
www.drugsincontext.com/biochemical-...
#AlkalinePhosphatase #Hypophosphatasia #RareDiseases #OrphanDrugs @me.pharmsky.app @moderation.medsky.network
@dhsc-answers.bsky.socialA question on #Cancer #OrphanDrugs tabled by Dr Scott Arthur on 14-07-2026 has been answered by Mr James Frith. https://questions-statements.parliament.uk/written-questions/detail/2026-07-14/18871
@gmj-georgia.bsky.socialNew analysis: of 814 FDA orphan drug approvals (2011–2023), only 29% received matching EMA authorisation with orphan status—and 33% were not authorised by EMA at all. Growing regula… #Drugaccess #EMA #FDA #Orphandrugs #Rarediseasepolicy #Drugaccess #EMA #FDA #Orphandrugs #Rarediseasepolicy htt…