P224 Effect of apitegromab on motor function at 36 months in patients with nonambulatory spinal muscular atrophy aged 2-12 years old

Neuronal degeneration and muscle atrophy contribute to functional decline in spinal muscular atrophy (SMA). Apitegromab inhibits the pro- and latent forms of myostatin, a negative regulator of skeletal muscle growth, directly targeting muscle atrophy. Improvements in motor function measures were observed with apitegromab in Type 2 and nonambulatory Type 3 SMA receiving nusinersen in the phase 2 TOPAZ study. The aim of the present analysis is to determine the effects of apitegromab (20 mg/kg) on muscle function as measured by HFMSE, RULM, and WHO motor development milestones at 36 months in patients with non-ambulatory SMA aged 2-12 years, the population that is the focus of the ongoing pivotal phase 3 SAPPHIRE trial.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: T. Crawford, B. Darras, J. Day, D. De Vivo, E. Mercuri, A. Nascimento, E. Mazzone, TOPAZ Study Team, A. Waugh, G. Song, R. Evans, J. Marantz Source Type: research