GM2 PFDD Meeting

Empowering Rare Voices
to Further Drug Development

Live Polling

Respond to polling questions in real time!

Go to PollEv.com/GM2PFDD and make your voice count!

Phone-In Live

Want to talk to us live during the discussion sessions?

Please call 1 703-844-3231 when prompted.

*Due to time restrictions, we may not be able to get to all phone calls.

Submit a Written Comment

Submit a comment below on the topics we will be discussing in advance of the EL-PFDD meeting. Comments should be submitted individually.

The comments collected may be shared during the GM2 EL-PFDD and may also be used in the final Voice of the Patient report (with identifying information removed).

If you are a parent or caregiver of a child with GM2 (Tay-Sachs or Sandhoff):

Which GM2 symptoms have the most significant impact on your child’s life?
How does GM2 affect your child on his/her best and worst days? Describe his/her best and worst days.
What specific activities is your child is unable to do at all or has difficulty doing because of GM2?
How have your child’s symptoms changed over time? How has his/her/your ability to cope with the symptoms changed over time?
What do you fear the most as your child progresses? What worries and frustrates you most about his/her condition?
What are you currently doing to manage your child’s GM2 symptoms?
How well do your current disease management interventions address your child’s most significant symptoms of GM2?
What are the most significant downsides to your child’s current interventions and how do they affect your daily life?
Short of a complete cure, what specific things would you look for in an ideal treatment for GM2?

If you are an adult living with GM2 (Tay-Sachs or Sandhoff):

Which GM2 symptoms have the most significant impact on your life?
How does GM2 affect you on your best and worst days? Describe your best and worst days.
What specific activities, that are important to you, that you cannot do at all or as fully as you would like because of GM2?
How have your symptoms changed over time? How has your ability to cope with the symptoms changed over time?
What do you fear the most as you get older? What worries and frustrates you most about your condition?
What are you currently doing to manage your GM2 symptoms?
How well do these disease management interventions address the most significant symptoms of your GM2?
What are the most significant downsides to your current interventions and how do they affect your daily life?
Short of a complete cure, what specific things would you look for in an ideal treatment for GM2?