P234 Effect of Apitegromab on pedi-cat and promis-fatigue questionnaire at 36-months in patients with spinal muscular atrophy

Spinal muscular atrophy (SMA) is characterized by neuronal degeneration and muscle atrophy. Apitegromab inhibits the pro- and latent forms of myostatin, a negative regulator of skeletal muscle growth and is being investigated in SMA, directly targeting muscle atrophy. The TOPAZ Phase 2 study of apitegromab showed improvements in muscle function and PEDI-CAT and PROMIS measures in patients with SMA Type 2 and nonambulatory Type 3, suggesting the potential of restoring muscle strength to improve patient/caregiver reported outcomes.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: T. Crawford, B. Darras, J. Day, J. Krueger, E. Mercuri, A. Nascimento, A. Pasternak, T. Duong, Topaz Study Team, L. Liu, M. Sadanowicz, S. Baver Source Type: research