Table 7 Factors of importance in PH1 medication development

Improve lab results (e.g., decreased oxalate)

N = 21; 75%

“But if it makes me feel better or makes those values [oxalate] decrease even

more, then…I’d be willing to try something else.”

Physical symptoms:

 • Reduce frequency and severity of kidney stones

 • Improve fatigue/energy levels

 • Decrease frequency of urination

 • Reduce/eliminate ulcers

N = 14; 50%

“The thought of not having to even go through a kidney stone, that would be incredible."

“If I got less kidney stones.”

“We’re definitely interested for any medications that can make her, help her lead a normal life without having, to have such frequent urination…”

“I would say reduce as many symptoms [ulcers] as possible.”

Prevent need for dialysis or kidney/liver transplantations

N = 6; 21%

"There is no cure, but I think if you can lessen the chances of having to be put on dialysis or get a transplant, I think that that is a very successful medication that I would love to have had."

Reduce need for increased fluid intake

N = 5; 18%

"If there was any way that it could reduce the amount of water they have to drink. Water is a four-letter word in our house, and it’s not fun. And trying to get a child to drink that much water every single day is so hard."

Allow for more flexibility in diet

N = 3; 11%

Reduce emotional impacts

N = 3; 11%

"I think those emotional impacts would come as a result of the other symptoms being lessened, let’s say fatigue or those aches and pains which correlate in their own way emotionally as a result of how extreme is the fatigue, how extreme is the pain."