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Final phase 3 trial analysis confirms recombinant ADAMTS13 (rADAMTS13) provides effective prophylaxis with fewer acute and subacute TTP events and better safety than plasma-based therapy in congenital TTP patients.
This phase 3 trial found that ecopipam reduced tic relapse and maintained symptom improvement in children and adolescents with Tourette syndrome, with mainly central nervous system side effects and no major weight or metabolic concerns.
Celcuity’s phase 3 VIKTORIA-1 trial reveals that gedatolisib, a broad-spectrum PI3K/mTORC1/2 inhibitor, significantly improves progression-free survival in hormone receptor-positive, HER2-negative advanced breast cancer patients post
Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.
