As I posted on the Neuren forum yesterday, following on from a Phelan McDermid focused Externally-Led Patient Focused Drug Development (EL-PFDD) meeting last November, resulting reports and a video can now be found online.
The meeting was hosted by CureSHANK and the Phelan-McDermid Syndrome Foundation and was modelled after the work of the FDA’s Patient-Focused Drug Development initiative, a systematic way of gathering patient and caregiver perspectives on their condition and on available treatments.
A similar EL-PFDD meeting was held for Rett syndrome last year, and there were 24 attendees from the FDA who listened to the testimonials and opinions of Rett patients and their families. In its recent review of trofinetide’s successful marketing application for Rett syndrome, the FDA included the parent/caregiver perspectives from the EL-PFDD meeting in their decision-making.
If you are interested in learning about Phelan-McDermid as well as its impact on those with the disorder and their parents/caregivers, I strongly urge you to have a look/read.
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