muSRK-015 builds Muscle Mass and Strength in Combination with Dystrophin Upregulation in a Mouse Model of DMD


Topic:

Translational Research

Poster Number: P160

Author(s):

Adam Fogel, PhD, Scholar Rock

Selective inhibition of myostatin activation with SRK-015 is a promising approach for safely building skeletal muscle and strength in neuromuscular disorders. We previously demonstrated in the SMNΔ7 model of SMA that enhancing the skeletal muscle with a selective inhibitor of myostatin, while concurrently addressing motor neuron defects with an SMN upregulator, can build muscle and promote strength, an approach now validated in a clinical setting. Here, we tested whether muscle defects in a DMD model may be addressed by a combination of two agents that target the muscle, muSRK-015P and a dystrophin corrector. Our data demonstrate that the combination treatment leads to muscle mass and strength increases in the tibialis anterior and gastrocnemius. These effects support the potential of selective myostatin inhibition with SRK-015 to build muscle mass and strength in moderately dystrophic muscle or in dystrophic muscle with partial dystrophin restoration.