The @fda.gov has approved the first gene therapy for children with Sanfilippo syndrome type A.

Link in the comments 👇

#ClinicalMondays #GeneTherapy #SanfilippoSyndrome #Pediatrics #OrphanDrug #FastTrack #BreakthroughTherapy

In case you missed it, our latest whitepaper provides a practical guide to closing the rare disease product timeline gap; evidence transportability, external control arms, sample-size preservation in real-world evidence, and where to invest early. buff.ly/rlXt6uf

#EUHTA #JCA #OrphanDrug #RWE