Table 7 Changes in impact on patients reported by caregivers after the start of the pabinafusp alfa trial

Physical function

Improved (n = 4): sleeping habits, decrease in falls, reduced snoring, ability to keep the mouth closed and breathe through the nose, exposure to germs, ability to move around, ability to ascend/descend stairs, kneel, athletic ability (n = 1 each)

Worsened (n = 3): wheelchair required, difficulty walking, difficulty running, difficulty kneeling, difficulty sitting cross-legged, difficulty rising from sitting (n = 1 each)

Activities of daily living

Improved (n = 4): eating independently (n = 2, I probed), using bathroom alone (n = 2, one probed), able to use chopsticks (unable previously), interest in phones, interest in picture books, improvements in bike riding, switching from diapers to underpants (n = 1 each)

Worsened (n = 2): ability to eat (n = 2 probed)

School/social life

Improved (n = 5): social interactions (n = 4, three probed), school life (n = 3, two probed), social independence, going to school daily (n = 1 each)

Emotional state

Improved (n = 3): calm/less anxiety (n = 3 probed), happier/normal, empathetic (n = 1 each)

Cognition/development

Improved (n = 2): self-expression, understanding speech, speaking ability, following directions (n = 1 each)