Grand Rounds – Dawn of a New Therapeutic Era for Spinal Muscular Atrophy

Jane Fan, MD

Associate Professor
UNC Child Neurology

Yael Shiloh-Malawsky, MD

Assistant Professor
UNC Child Neurology

Objectives:

1. Review the genetic mechanism of SMA disease process
2. Review clinical presentation and natural history of the disease
3. Discuss of newly approved treatment of SMA – Nusinersen/Spinraza, mechanism and clinical trials results
4. Discuss new developments in gene therapy for SMA and future of treatment of SMA

321 MacNider Hall
Thursday, December 14, 2017, 8:00 AM

Sponsored by the School of Medicine of The University of North Carolina at Chapel Hill.