Recent regulatory activity underscores progress in therapeutics for rare and difficult-to-treat diseases. Ionis Pharmaceuticals secured FDA approval for Dawnzera, a prophylactic antisense therapy for hereditary angioedema, demonstrating significant reductions in swelling attack rates across global trials. Meanwhile, Stealth BioTherapeutics' NDA resubmission for its Barth syndrome treatment, elamipretide, received expedited FDA review with a planned decision this September amid active patient and scientific community advocacy. These approvals and reviews highlight renewed momentum in rare disease therapeutics, driven by novel mechanisms and unmet clinical needs. Additionally, Agendia's expanding MammaPrint registry data reinforces the clinical utility of genomic risk stratification in breast cancer, guiding chemotherapy decisions across risk groups.