The Facioscapulohumeral Muscular Dystrophy-Health Index: Development and evaluation of a disease-specific outcome measure.
Anika VarmaJennifer WeinsteinJamison SeaburySpencer J RoseroCharlotte EngebrechtEllen WagnerChristine ZizziElizabeth A LuebbeNuran DilekMichael P McDermottJohn KisselValeria SansoneChad HeatwolePublished in: Muscle & nerve (2023)
The FSHD-HI provides researchers and clinicians with a reliable and valid mechanism to measure multifaceted disease burden in patients with FSHD. The FSHD-HI may facilitate quantification of therapeutic effectiveness, as demonstrated by its use as a secondary and exploratory measure in several clinical trials.