disease treatment
SpringWorks Therapeutics Secures FDA Approval for Gomekli to Treat NF1-Related Tumors
SpringWorks Therapeutics, Gomekli, mirdametinib, FDA approval, neurofibromatosis type 1 (NF1), plexiform neurofibromas (PN), rare disease treatment, MEK inhibitor
Narsoplimab Demonstrates Significant Survival Benefit in Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy
Narsoplimab, Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA), Lectin Pathway Inhibition, MASP-2 Inhibitor, Rare Disease Treatment, Overall Survival Improvement
Translarna by PTC Therapeutics: Awaiting Another EU Regulatory Review for Muscular Dystrophy Treatment
PTC Therapeutics, Translarna, muscular dystrophy, EU review, regulatory approval, Duchenne muscular dystrophy (DMD), European Medicines Agency (EMA), ataluren, rare disease treatment.