Spinal muscular atrophy treatment with disease-modifying therapy

The main goal of disease-modifying therapy for patients with spinal muscular atrophy is to slow the development of the symptoms. In some rare cases, the progression of the disease might even be stopped for some time, which is certainly better compared to the entire absence of treatment. Before starting any treatment, medical practitioners try to obtain as much information about the course of the disease and the patient’s current condition. As a result, the patient should undergo numerous medical diagnostic procedures and lab tests. Currently, the FDA approves two main medications for a disease-modifying therapy for spinal muscular atrophy: Risdiplam (Evrysdi) and Nusinersen (Spinraza). Both drugs can help to increase the production of SMN protein, which is necessary for the normal functioning of motor neurons (nerve cells responsible for controlling movement). Also, both medications can be used for adults and childer over two months old. Spinraza is a liquid drug that is most often delivered via injections in the spinal canal. As a result, medication mixes up with cerebrospinal fluid and causes a complex effect on the whole organism. Evrysdi is usually prescribed in an oral-administer form, which is more comfortable, but might be more costly.