Browsing Tag
enzyme replacement therapy
4 posts
Can fewer infusions improve adherence in Fabry disease enzyme replacement therapy?
EU approves monthly Elfabrio dosing for Fabry disease. Discover how reduced infusion frequency could reshape enzyme therapy and treatment burden.
JCR Pharmaceuticals completes global Phase III enrollment for JR-141 targeting Hunter syndrome’s neurological symptoms
JCR Pharmaceuticals completes global Phase III enrollment for JR-141, targeting Hunter syndrome CNS symptoms with its BBB-penetrating enzyme therapy.
JR-446 developed by JCR Pharmaceuticals secures orphan drug designation from the European Commission for MPS IIIB
The European Commission has granted orphan drug status to JR-446 for MPS IIIB, supporting JCR Pharmaceuticals’ global rare disease expansion.
Orphan Technologies starts OT-58 trial for homocystinuria treatment breakthrough
Swiss pharmaceutical company Orphan Technologies has begun dosing patients in a Phase 1/2 trial (CBS-HCY-01) to assess the…