Functional Outcome Measures to Optimize Drug Development in Spinal and Bulbar Muscular Atrophy
2024; Lippincott Williams & Wilkins; Volume: 103; Issue: 12 Linguagem: Inglês
10.1212/wnl.0000000000210088
ISSN1526-632X
AutoresSpencer B. Huggett, Andrew T.N. Tebbenkamp, Carlo Rinaldi, Dipa Jayaseelan, Luca Zampedri, Lorenzo Blasi, Andrea Fortuna, Abdullah Alqahtani, Angela Kokkinis, Julia R. Dahlqvist, Silvia Fenu, Eleonora Cavalca, Alessandro Bertini, Caterina Mariotti, Christopher Grunseich, T. Kawase, Yoshiyuki Kishimoto, Shinichiro Yamada, Masahisa Katsuno, Pietro Fratta, Amelia Conte, Mario Sabatelli, Gianni Sorarù, John Vissing, Minsung Kang, Jin‐Sung Park, Davide Pareyson, Vissia Viglietta,
Tópico(s)Genetic Neurodegenerative Diseases
ResumoSpinal and bulbar muscular atrophy (SBMA) is a rare, slowly progressive, and debilitating disease without effective treatments available. Lack of reliable biomarkers and sensitive outcome measures makes clinical research conduct challenging. The primary objective of this study was to identify clinically meaningful and statistically sensitive outcome measures enabling the evaluation of therapeutic interventions in late-stage clinical trials.
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