Acadia Pharmaceuticals has launched DAYBUE STIX for Rett syndrome in the U.S. Read what this means for adoption, tolerability, and market strategy.
Affinia Therapeutics has cleared AFTX-201 for a Canadian BAG3 DCM trial. Read why the gene therapy’s design and risks matter now.
Soligenix published HyBryte versus Valchlor data in CTCL. Read what it changes for approval, adoption, and competitive positioning.
CHMP backs paediatric lomitapide for HoFH. Read what this means for clinicians, regulators, and rare disease access across Europe.
Drug Farm won FDA orphan drug designation for DF-003 in ROSAH syndrome. Read what this rare disease milestone could change next.
Shionogi’s S-606001 enters global Phase 2 as the first oral GYS1 inhibitor tested as an add-on to ERT in late-onset Pompe disease. Read the analysis.
GenSight Biologics begins treating ND4-LHON patients under France’s AAC compassionate access program. Analyse what REVISE and AAC mean for the regulatory pathway.
KalVista’s sebetralstat pediatric trial data heads to Madrid. What it means for the HAE on-demand market and a 2026 FDA filing. Read the analysis.
IntraBio files the first-ever FDA application for an A-T therapy. Analyse the Phase III evidence, regulatory pathway, and what approval would mean.
Sarepta’s ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.