Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported Outcome Measure.
Jamison SeaburyAnika VarmaJennifer WeinsteinSpencer J RoseroCharlotte EngebrechtShaweta KhosaChristine ZizziEllen S WagnerDanae AlexandrouBrittany L CohenNuran DilekJohn M HeatwoleDavid R LynchCourtney C ParkMcKenzie WellsSub H SubramonyChad R HeatwolePublished in: Neurology. Clinical practice (2024)
Initial evaluation of the FACR-HI supports its content validity, test-retest reliability, and construct validity as a caregiver-reported outcome measure for assessing how pediatric individuals with FRDA feel and function. The FACR-HI provides a potential mechanism to quantify changes in multifactorial FRDA disease burden during future clinical trials.