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Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says
Zachary Brennan (Endpoints News) February 27, 2027 The FDA will increasingly use accelerated approval for gene therapies aimed at rare...
Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine
Demaris Mills (Forbes) January 12, 2024 The 2023 Oligonucleotide Therapeutics Society meeting drew 800 attendees to Barcelona, a clear...
Pilot launched to support children with rare conditions to access personalised therapies
Genomics England November 22, 2023 New programme announced to develop a pathway for children with rare conditions to access therapies...
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...