The Feasibility of Personalized Endpoints in Assessing Treatment Outcomes for Rare Diseases: A Pilot Study of Goal Attainment Scaling in SCN2A-Associated Developmental Epileptic Encephalopathy

• DOI: 10.1016/j.jval.2024.12.004
• Publication Date: 2024-12-27T16:51:28Z
• Authors: Gunes Sevinc; Kari Knox; Michelle George; Lindsey Evans; Ariela Kaiser; Katherine Charlot...; Leah Schust Myers; Natasha N. Ludwig; Mary Wojnaroski; Gabrielle Conecker; JayEtta Hecker; Jenny Downs; Chere A.T. Chapman; Anne T. Berg
• Keywords: Goal Attainment Scaling