What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists
October 12, 2023 I first saw Terry Horgan as a patient. I was filling in as the pediatric pulmonary specialist in the UMass-Duchenne...
What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists
‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study
All of Us and the Promise of Precision Medicine:
MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE
Regulatory Pathway: Where We Are Today
Individualised medicines offer hope for children with a rare disease
Potential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic