What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers
FDA Law Blog
MARCH 13, 2023
The FDA took an early lead in the evolution of this movement when they launched the Patient Focused Drug Development (PFDD) initiative in 2012. One family’s daughter was the first U.S. Other families have had to try and get access in Milan, Italy, where a gene therapy for MLD has already been approved.
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