In these studies, the addition of reldesemtiv to either nusinersen or SMN-C1 resulted in a leftward shift of the force-frequency curve, indicating an increase in calcium sensitivity of the muscle at submaximal stimulation frequencies and confirming the efficacy of fast skeletal muscle activation in muscle in conjunction with SMN upregulators. Augmentation of muscle force at submaximal stimulation frequencies may be relevant to activities such as breathing and walking. These data suggest that treatment with reldesemtiv in combination with an SMN upregulating therapy such as nusinersen or SMN-C1, may complement and further improve muscle function in SMA.
Nusinersen is an approved anti-sense oligonucleotide therapy for SMA that increases production of Survival Motor Neuron (SMN) protein. SMN-C1 is a small molecule therapy that increases SMN protein expression, similar to risdiplam, an investigational treatment in development for SMA.
Publicerat den: 25 jun, 2019
Uppdaterad den: 28 jun, 2019