P227 MANATEE: GYM329 (RO7204239) in combination with Risdiplam treatment in patients with spinal muscular atrophy (SMA)

Risdiplam (EVRYSDI ®) is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved for the treatment of spinal muscular atrophy (SMA) in more than 90 countries worldwide. GYM329 is an investigational, recycling and antigen-sweeping monoclonal anti-myostatin a ntibody (myostatin is a negative regulator of skeletal muscle growth). In preclinical studies, the combination of GYM329 with an SMN2 splicing modifier demonstrated improved muscle size and strength compared with SMN2 splicing modifier treatment alone.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: T. Duong, B. Darras, J. Morrow, F. Muntoni, L. Servais, M. Rabbia, M. Gerber, H. Kletzl, E. Gaki, S. Fletcher, R. Scalco, K. Wagner, E. Mercuri Source Type: research