Patient-Reported Impact of Symptoms in Spinal and Bulbar Muscular Atrophy.
Abdullah AlqahtaniAngela KokkinisChristine ZizziNuran DilekKenneth H FischbeckChad R HeatwoleChristopher GrunseichPublished in: Neurology. Clinical practice (2023)
There are a diversity of themes that are important to patients with SBMA. These themes have a variable level of importance to the population with SBMA and represent clinically meaningful outcome measures for future therapeutic interventions.