Browsing Tag
lysosomal storage disorder
2 posts
What is Niemann–Pick disease and why is this rare genetic disorder so hard to treat?
Discover Niemann–Pick disease, a rare genetic disorder affecting lipid metabolism. Learn about its types, symptoms, causes, diagnosis, and latest treatments.
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.