The Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting for Phelan-McDermid syndrome (PMS) was held on November 8, 2022 (recording below).  
Through December 7, 2022, we encourage those caring for loved ones living with PMS to continue submitting comments using the form below.
All comments will be included in the final Voice of the Patient report produced from this EL-PFDD project.
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Recording & Comments:
EL-PFDD Meeting for PMS

Submit a Written Comment

Submit a comment below on the topics we are discussing at the EL-PFDD meeting. Comments should be submitted individually.

  • Of all the symptoms of Phelan-McDermid syndrome, which 1-3 symptoms have the most significant impact on you or your loved one’s life?

  • How does Phelan-McDermid syndrome affect you or your loved one on best and on worst days? Describe your best days and your worst days.

  • Are there specific activities that are important that you or your loved one cannot do at all or as fully as you would like because of Phelan-McDermid syndrome?

  • How has your loved one’s ability to cope with the symptoms changed over time?

  • What are you currently doing to manage your loved one's PMS symptoms?

 

  • How well do these treatments address the most significant symptoms and health effects of PMS?