What Kyowa Kirin’s Crysvita dosing change reveals about rare disease treatment individualisation
Rare disease dosing is becoming more personalised. Kyowa Kirin’s Crysvita update tests how far flexibility can improve adult XLH care.
Could Entos Pharmaceuticals’ Fusogenix platform open a real path to treatment in L-CMD?
Entos Pharmaceuticals and the L-CMD Research Foundation are targeting a curative L-CMD therapy. Read what this could change for rare disease treatment.
Acadia expands DAYBUE delivery formats for Rett syndrome with powder-based STIX
Acadia Pharmaceuticals has announced U.S. Food and Drug Administration approval for DAYBUE STIX (trofinetide) for oral solution, a new powder-based formulation of its existing drug DAYBUE used in the treatment of Rett syndrome. The approval covers use in adult and pediatric patients two years and older and is based on bioequivalence data, not new clinical […]