Articles & PodcastsWhat We Owe Terry Horgan: Reflections from Providers, Family, and ScientistsOctober 12, 2023 I first saw Terry Horgan as a patient. I was filling in as the pediatric pulmonary specialist in the UMass-Duchenne...
Illustrated Examples‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
Seminar SeriesPotential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic
