Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD).

Anika VarmaJennifer WeinsteinJamison SeaburySpencer RoseroNuran DilekJohn HeatwoleCharlotte EngebrechtShaweta KhosaKaitlin ChungAsif PakerAmy WooGregory BrooksChan BealsRohan GandhiChad Heatwole

Published in: Orphanet journal of rare diseases (2024)

There are many patient-relevant symptoms and themes that contribute to disease burden in individuals with ALD. These symptoms, identified by those having ALD, present key targets for further research and therapeutic development.

Keyphrases

patient reported
sleep quality
case report
risk factors
depressive symptoms