Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease.
Ryan Haumschild, PharmD, MS, MBA, and Mary Pak, MD, FACP, consider FDA-approved gene therapies for hemophilia, spinal muscular atrophy, and Duchenne muscular dystrophy, exploring their genetic foundations, the FDA approval process, and considerations for therapeutic value and cost.
A panel of experts analyze the impact of prefilled syringes on workflows across diverse patient populations, emphasizing cost savings, patient safety, and interdisciplinary collaboration.
Explore the multifaceted impact of desmoid tumors on patients’ physical, functional, and psychosocial well-being as experts emphasize effective symptom management and quality-of-life enhancements.