@pharmaphorum.bsky.social#AstraZeneca has already filed for approval of its therapy for ultra-rare disease #hypophosphatasia (HPP), after missing the mark in a phase 3 trial, but reckons the totality of the data could get the drug over the line with a broad label.
@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
