MK-51

Phase 3 CAHtalyst pediatric study demonstrates that crinecerfont (CRENESSITY) effectively reduces glucocorticoid dosing while maintaining or improving androstenedione levels in children with classic congenital adrenal hyperplasia.

The FDA has granted orphan drug designation to Sanofi's rilzabrutinib for warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), two rare conditions with no currently approved treatments.

Innovent Biologics' SYCUME® (teprotumumab N01) has received NMPA approval as China's first IGF-1R monoclonal antibody for treating thyroid eye disease, ending a 70-year treatment innovation drought.

The FDA has approved Crenessity (crinecerfont) as an adjunctive treatment for congenital adrenal hyperplasia (CAH) in adults and children, marking a significant advancement.

Five-year follow-up data from Study 101 supports the role of delandistrogene moxeparvovec (Elevidys) in stabilizing or slowing Duchenne muscular dystrophy (DMD) progression.