Experimental Treatments for SMA
Overview
Spinal muscular atrophy (SMA) is an inherited neurodegenerative disorder characterized by progressive muscle weakness. SMA patients do not produce enough of a protein called survival motor neuron (SMN), due to a mutation in the SMN1 gene. Another gene, SMN2, also can produce some SMN protein, but it is less stable.
A number of experimental therapies are in the pipeline for the potential treatment of SMA. These include treatments aimed at increasing SMN protein levels, enhancing residual SMN function, and compensating for its loss. Some of the therapies currently in development for SMA are summarized below.
Apitegromab
Apitegromab (SRK-015) is an experimental muscle-directed infusion therapy being developed as an add-on treatment to improve motor function in people with SMA. Rather than addressing the root cause of the disease, apitegromab is designed to boost patients’ muscle strength by targeting a muscle protein called myostatin.
