Table 3.

Representative quotes about desired new treatments

Example quotes from participant responses
“If they could have a less-restricted diet, that would be amazing. […] they could just fit in more with their peers and eat food a lot more similar to what they’re eating and have options at a restaurant besides French fries. […]. So that they could enjoy being a kid.” (Caregiver 300-012-2)
“That’s like my dream is to not have to be on a diet. Not have to take formula, you know, or have a once-a-week injection or once a month, or twice a week. […] If I couldn’t come off of the diet totally, if it doubled my protein intake or you know halved the amount of formula that I had to take, I would consider that a worthwhile improvement.” (Adult patient 300-008-3)
“I guess if I had more leeway in my diet, it would be a little more relieving. […] It would obviously make a difference because the less grams of protein that you’re allotted, the more strict you have to be. But if you can offset that and say, even two grams more, […] it doesn’t seem like a lot, but it is a lot, could help alleviate some of those dietary restrictions.” (Adult patient 300-005-3)
“More energy, weight gain. […] Happier. […] Fewer days of fatigue, definitely. But also not having to be on a diet.” (Pediatric patient 300-013-1)