Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to three for all forms of disease.
Spinraza was first approved in 2016 as a therapeutic option specifically for SMA. Then in 2019 Zolgensma was approved. This is a gene therapy which is meant to treat young children.
Roche has been working on a new therapeutic option called risdiplam. It’s unique in the fact that its an oral therapy, meaning patients don’t have to deal with injections. Spinraza can only be administered through the spinal cord, directly into the cerebrospinal fluid. Zolgensma has also been administered this way in some trials. An oral therapy means patients don’t have to face this invasive procedure.
You can read more about this therapy here.