Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein. While current standards of care,…

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A Potential Turning Point: CAR T-Cell Therapy Shows Long-Term Survival in Advanced Multiple Myeloma

At the recent American Society of Clinical Oncology (ASCO) annual meeting, researchers unveiled remarkable long-term results for ciltacabtagene autoleucel (cilta-cel; Carvykti), a CAR T-cell therapy, in patients with heavily pretreated…

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