Spinal muscular atrophy (SMA) is one of the leading causes of death in infants 
related to the degeneration of neurons. Currently, there are no curative 
treatment options for SMA, and many options available may not be feasible. This 
review presents the background, clinical studies, and indications for the use of 
Risdiplam in treating SMA. SMA causes a decrease in the production of survival 
motor neuron proteins (SMN) and current treatments target to increase the 
expression of SMN. Risdiplam is the first and only oral medication to be 
approved to treat SMA. As an SMN2 splicing modifier, it has provided stronger 
systemic therapies than previous intrathecal and gene replacement therapies. 
There have been many efforts to treat SMA with multidisciplinary approaches. 
These include intrathecal injections to gene replacement therapies. However, 
these have been faced with limitations such as reaching a good therapeutic dose 
in systemic tissues, route of administration, and price. Risdiplam is currently 
the only orally administered drug approved by the FDA for the treatment of SMA. 
It not only provides a good therapeutic window to systemic tissues but allows 
for a non-invasive approach in infants. Further investigation and comparison on 
the safety profile of Risdiplam due to its broader systemic effect should be 
considered with other available therapies.