PTC SEPHIENCE: A New PKU Treatment Approved
FDA-approved SEPHIENCE (sepiapterin), an oral drug, treats PKU in patients 1 month+. SEPHIENCE aims to be the future standard of care.
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Apellis' EMPAVELI: First Treatment for Rare Kidney Diseases
FDA-approved EMPAVELI (pegcetacoplan) for C3G & IC-MPGN in patients 12+. Shows 68% proteinuria reduction, kidney function stabilization, and C3 deposit clearance. A transformational therapy for 5,000 US patients.
Celcuity's Gedatolisib Shows Unprecedented Breast Cancer Trial Results
Phase 3 VIKTORIA-1 trial shows gedatolisib triplet slashed disease progression risk by 76% in HR+/HER2- advanced breast cancer, with favorable safety.