Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregivers.
Marielle G ContesseRebecca J WoodsMindy LefflerSilvia PradesJulie GreenfieldAndrea ComptonJeffrey B CarrollPublished in: Therapeutic advances in rare disease (2024)
This study found that there are symptom domains that are relevant across the DRPLA population, but there is heterogeneity within each domain based on the age of symptom onset and disease stage, which has implications for clinical trial design.