The Rare360 Editorial TeamFeb 192 minFDA Extends Priority Review Period for Kresladi/ RP-L 201 in Treating Severe Leukocyte Adhesion Deficiency-I (LAD-1)Explore the FDA's extension of the priority review period for Kresladi, a groundbreaking gene therapy for severe LAD-1
The Rare360 Editorial TeamJan 183 minMilestone in Gene Therapy: Casgevy Approved for Transfusion-Dependent Beta ThalassemiaExplore the FDA's groundbreaking approval of CASGEVY™ the treatment of transfusion-dependent beta-thalassemia (TDT).
The Rare360 Editorial TeamDec 12, 20233 minNew Horizon in PNH Treatment: FDA Approves First Oral DrugExplore the latest in PNH treatment with the FDA's groundbreaking approval of iptacopan (Fabhalta).
The Rare360 Editorial TeamNov 6, 20233 minNew Hope for Pediatric Patients: Bosutinib's Approval in Treating Chronic Phase Ph+ CMLLearn about Ph+ CML, its phases, and the FDA's approval of bosutinib for pediatric patients with chronic myeloid leukemia.
The Rare360 Editorial TeamNov 1, 20233 minAssessing Safety and Effectiveness of Damoctocog Alfa Pegol in Hemophilia A PatientsHEM-POWR study shows damoctocog alfa pegol's promise, reducing bleeding and improving joint health in hemophilia A patients.
The Rare360 Editorial TeamSep 21, 20232 minLate-Onset Pompe Disease: New Clinical Insights Pave the Way for Enhanced Quality of LifeA groundbreaking study offers hope to the LOPD community by revealing crucial clinical markers that could transform diagnosis and treatment.