Save the Date! September 25, 2020!
What: CureDRPLA and the National Ataxia Foundation EL-PFDD
Where: Online. Due to Covid-19, our meeting will be virtual. Link to be provided closer to the date.
When: 10:00AM-3PM EST
Who: All DRPLA Patients and their Caregivers. This meeting can be accessed virtually anywhere in the world. Please attend to share your perspective.
More Details at:. https://www.eventbrite.com/e/119165841239
What is an EL-PFDD? An externally-led patient-focused drug development (EL-PFDD) meeting can give FDA and other key stakeholders an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight during drug development.
Still confused? Watch our informational webinar to learn more about what this meeting means, how the process will work, and how you can get involved. Link here: https://www.youtube.com/watch?time_continue=19&v=b0KP4GGY4dk&feature=emb_logo
Any questions please contact email@example.com
11:20AM EST – TOPIC 1 – LIVING WITH SCA/DRPLA: SYMPTOMS AND DAILY IMPACT
1. For the person who is affected with SCA/DRPLA, please identify 1-3 symptoms that have the most significant impact:
a) Currently which symptoms most affect the person with SCA/DRPLA?
b) Which symptoms were the most significant at other times for the person with SCA/DRPLA?
c) When looking back after being diagnosed with SCA/DRPLA what were the first symptoms the person with SCA/DRPLA experienced?
2. How have symptoms changed or progressed over time?
3. How does SCA/DRPLA affect the person with SCA/DRPLA on the best days?
4. How does SCA/DRPLA affect the person on the worst days?
5. Are there specific activities that are important to the person with SCA/DRPLA that cannot be done at all or as fully as you would like because of SCA/DRPLA?
a) How does it affect life activities (school/work, abilities, relationships, self-sufficiency, living situation, activities, etc.)?
b) If the person with SCA/DRPLA could do one activity that they currently are unable to, what would it be?
6. What worries you the most as you get older for either yourself or for the person with SCA/DRPLA?
1:35PM EST – TOPIC 2 – PERSPECTIVE ON CURRENT AND FUTURE APPROACHES TO TREATMENT
1. What are you currently doing to manage your SCA/DRPLA symptoms?
a) Which specific SCA/DRPLA symptoms do the treatments address?
b) How has this treatment regimen changed over time and why?
2. How effective have these treatments been for you/the affected individual?
a) How well do these treatments improve the ability to do specific activities that are important in daily life?
3. What are the most significant downsides to current treatments and how do they affect daily life? (Examples of downsides may include bothersome side effects, going to the hospital for treatment, etc.)
4. Short of a complete cure, what specific things would you want for an ideal treatment for SCA/DRPLA? When considering a new treatment, what factors are important to you?
a. What questions/data/information would you want to consider?
b. Imagining that there were multiple treatment options approved, what factors into your decision on which treatment to choose?