This meeting is about helping the FDA and researchers understand what life with Pitt Hopkins is really like: the daily challenges, the treatments that help, the needs that remain unmet, and what meaningful improvements could look like.

We cannot assume someone else will tell our story. The more families who participate, the more complete a picture we can provide of life with Pitt Hopkins.

How Can You Participate?

• Attend the meeting. Your presence helps demonstrate the strength and commitment of our community.
• Speak during the open forum. Families and patient representatives will have opportunities to share their experiences during facilitated discussions.
• Prepare your story. Think about the biggest challenges your loved one faces, what treatments you have tried, and what you hope future treatments could accomplish.
• Encourage other families. Share this opportunity and help us bring as many voices together as possible.

You do not need to be a medical expert or prepare a formal speech. You are the expert on your family’s experience. Even sharing one personal example can make a difference.

What Will We Discuss?

The meeting will include two major discussion topics. Review the questions below and consider which are most important to your family. You do not need to answer every question.

Topic 1: Health Effects and Daily Impacts

Help the FDA understand what living with Pitt Hopkins looks like every day.

1. What are the one to three symptoms or challenges that have the biggest impact on your loved one’s life?
2. What important activities are difficult or impossible because of Pitt Hopkins?
3. What does a good day look like? What does a bad day look like?
4. How have symptoms and needs changed over time? Is the condition well managed today?
5. What worries you most about Pitt Hopkins and your loved one’s future?

Topic 2: Current Approaches to Treatment

Help identify what works, what is missing, and what future treatments should accomplish.

1. What medications, therapies, or other approaches are you currently using to manage symptoms?
2. How has your loved one’s treatment plan changed over time, and why?
3. How well do current treatments help with the most significant symptoms? What challenges remain?
4. What are the downsides of current treatments, and how do they affect daily life?
5. What would an ideal treatment accomplish, short of a complete cure? What improvement would make the biggest difference in your loved one’s life?

A Few Things to Remember

• Be specific. Real-life examples help others understand your experience.
• Be honest. Share what works, what doesn’t, and what remains difficult.
• Focus on meaningful improvements. What would make life better for your loved one and your family?
• Don’t feel pressured to answer everything. Share what matters most to you. Attending and listening are valuable, too.

Let’s Make Our Voices Heard

We know families are busy balancing work, caregiving, medical appointments, and everyday life. We understand that participating takes time and effort.

But this is an opportunity to help shape the future of Pitt Hopkins research and treatment development.

Please plan to attend on November 13, share this opportunity with other families, and consider adding your voice to the discussion.

One family’s story matters. Together, we can help the FDA better understand the needs of the entire Pitt Hopkins community.

Bring your experience. Bring your perspective. Bring your voice.

Together, let’s help move Pitt Hopkins research forward.