Erythropoietic protoporphyria: time to prodrome, the warning signal to exit sun exposure without pain-a patient-reported outcome efficacy measure.

D WensinkJ G LangendonkJ R OverbeyM BalwaniE J E Van BroekhovenM A E M WagenmakersJ H P WilsonK WheedenH NaikRobert J Desnick

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2021)

EPP patients reported substantial improvements in TTP during afamelanotide treatment. TTP could provide a safer, PRO-based efficacy endpoint for assessing future EPP treatments.

Keyphrases

patient reported outcomes
end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
chronic pain
prognostic factors
peritoneal dialysis
pain management
neuropathic pain
spinal cord injury
spinal cord
smoking cessation
patient reported
replacement therapy