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 GSD1a EL-PFDD and may also be used in the final Voice of the Patient report (with identifying information removed).

Topic 1 - GSD1a Symptoms & Daily Impact

Of all the health effects of GSD1a, which 1-3 symptoms have the most significant impact on you or your loved one's life?
Examples may include hypoglycemia, stomach aches, nausea, exhaustion, anxiety, fear, etc.

How does GSD1a affect you or your loved one on best and on worst days?

How have your or your loved one's symptoms changed over time? How has the ability to cope with the symptoms changed over time?

Are there specific activities that are important to you or your loved on that you/they cannot do at all or as fully as  because of GSD1a?
Examples of activities may include sleeping through the night, participation in sports or social activities, going away to college, travel, etc.

What do you fear the most as you or your loved one gets older? What worries you the most about your or your loved one's condition?

Topic 2 - Current Approaches to GSD1a Treatments

What are you currently doing to manage your or your loved one's GSD1a? Examples may include over-the-counter products, prescription medications and other therapies including non-drug therapies such as diet modification or emotional support.

How well do these treatments treat the most significant symptoms and health effects of GSD1a? For example, how well do your treatments improve your ability to do specific activities or improve your overall quality of life?

What are the most significant downsides of your or your loved one's current treatments and how do they affect daily life? Examples of downsides may include going to the hospital or clinic for treatment, time devoted to treatment, side effects of treatment. 

Short of a complete cure, what specific things would you or your loved one look for in an ideal treatment for GSD1a? Think about what you would consider to be meaningful imporovement (for example, fewer hypoglycemic episodes, less cornstarch, more time between cornstarch doses, weight loss, less anxiety and stress, more energy, less diet restrictions, sleep, etc.)

What factors would be important in deciding whether to participate in a new research trial?