Behind the Science: Behind Updates on Advances for Rare Disease Day

In recognition of the recent celebration for Rare Disease Day, a number of experts in the care of rare diseases spoke to some of the advances and challenges experienced by the field in the past few years. They include: Hong Sun, MD, PhD, on the importance of Rare Disease Day; Sabrina Paganoni, MD, PhD, on the incremental advances being made in ALS drug development; Bruce Cree, MD, PhD, on the need for more patient education on neuromyelitis optica spectrum disorder; James Howard, MD, on the potential for combination approaches to generalized myasthenia gravis; and Tahseen Mozaffar, MD, on the potential of gene therapies for Pompe disease.