12/22/2025
Clinical trials are how we bring new treatments forward for Phelan-McDermid syndrome, but they only work when the data is protected.
Families participating in clinical trials are strongly discouraged from sharing personal trial experiences, including side effects, perceived changes, videos of improvements, or comparisons. This includes sharing on social media, with potential participants, or even with extended family and friends. Even well-intentioned sharing can introduce bias, lead families to guess whether their child is receiving treatment or placebo, and compromise the integrity of a study.
In other rare disease communities, this has had a negative impact and, in some cases, shut down trials, even when treatments were helping patients.
It is okay to share logistical support, such as travel tips or general praise for trial sites and staff. Any concerns about a participant's experience should be discussed directly with the study team or your loved one's care team.
Protecting trial integrity today helps ensure effective treatments can be proven, approved, and made available to our entire community.
🔗 Learn more about what can and cannot be shared by visiting the link in the comments.
