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EL-PFDD Meeting for PMS &
PMS Voice of the Patient Report

The Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting for
Phelan-McDermid syndrome (PMS) was held on November 8, 2022 (recording below).

The PMS Voice of the Patient Report is now also complete. This report compiles and summarizes the content of the EL-PFDD meeting. It was submitted to the FDA in March 2023, along with Additional Patient/Caregiver Comments submitted online (documents below). This report will be utilized by the FDA, industry, researchers, and other stakeholders as important context in development of treatments to meet the most urgent needs of the PMS community. 

and Additional Patient/Caregiver Comments

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EL-PFDD Recording

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