The Sisters' Hope Foundation externally-led patient focused drug development (EL-PFDD) session will be held on Friday June 20th, 2025. Information and insights gained during this meeting will be summarized in the "Voice of the Patient" report that will be published for public use. Register to participate in person or virtually through Zoom.

Register here!

Agenda
1:00 pm - Welcome: Sisters’ Hope Foundation
1:05 pm - Welcome: FDA Representative
1:10 pm - ALSP Clinical Overview: Clinician
1:20 pm - Introduction and Meeting Overview
1:25 pm - Remote Demographic Polling
Session 1: Health and Daily Effects of Living with ALSP
1:30 pm - Panelist Testimonials
2:00 pm - Moderated Audience Discussion and Remote Polling
3:00 pm - BREAK
Session 2: Current Approaches and Future Preferences to Treating ALSP
3:15 pm Panelist Testimonials
3:45 pm - Moderated Audience Discussion and Remote Polling
4:45 pm - Meeting Summary
4:55 pm - Next Steps and Closing

Date: Friday, June 20th, 2025
Time: 1pm-5pm
Venue: Live! Casino & Hotel, 900 Packer Avenue, Philadelphia, PA

Please contact Erin Sullivan at erin@sistershopefoundation.org with any questions.

TOPIC 1 QUESTIONS:

1. Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact on your life?

2. What specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition? Examples of activities may include sleeping through the night, daily hygiene, participation in sports or social activities, intimacy with a spouse or partner, etc.

3. As it relates to your condition, what does a good day look like? What does a bad day look like?

4. How have your condition and its symptoms changed over time?

5. Would you define your condition today as being well managed?

6. What worries you most about your condition?

TOPIC 2 QUESTIONS:

1. What are you currently doing to help treat your condition or its symptoms? Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modification.

2. How has your treatment regimen changed over time, and why?

3. How well does your current treatment regimen treat the most significant symptoms of your disease? For example, how well do your treatments improve your ability to do specific activities? How well have these treatments worked for you as your condition has changed over time?

4. What are the most significant downsides to your current treatments, and how do they affect your daily life? Examples of downsides may include going to the hospital or clinic for treatment, time devoted to treatment, restrictions on driving, etc.

5. What considerations would encourage or discourage your participation in a clinical trial?

6. Short of a complete cure, what specific things would you look for in an ideal treatment for your condition?

7.  What do you consider the most beneficial functional improvements if you could regain what you have lost or anticipate losing?