Thomas O Crawford, Basil T Darras, John W Day, Sally Dunaway Young, Tina Duong, Leslie L Nelson, Doreen Barrett, Guochen Song, Sanela Bilic, Shaun Cote, Mara Sadanowicz, Ryan Iarrobino, Tiina J Xu, Janet O'Neil, José Rossello, Amy Place, Nathalie Kertesz, George Nomikos, Yung Chyung
BACKGROUND AND OBJECTIVES: Currently approved therapies for spinal muscular atrophy (SMA) reverse the degenerative course, leading to better functional outcome, but they do not address the impairment arising from preexisting neurodegeneration. Apitegromab, an investigational, fully human monoclonal antibody, inhibits activation of myostatin (a negative regulator of skeletal muscle growth), thereby preserving muscle mass. The phase 2 TOPAZ trial assessed the safety and efficacy of apitegromab in individuals with later-onset type 2 and type 3 SMA...
March 12, 2024: Neurology