Spinal muscular atrophy impairs an individual’s ability to control their muscle movements. It is an inherited condition, and currently, there is no cure. However, treatments available can help manage spinal muscular atrophy. Also, many experimental therapies for treating the condition are in development. Doctors usually prescribe treatments based on the individual’s age, overall health, and the type of spinal muscular atrophy they have. In this article, let’s discuss popular treatment options for spinal muscular atrophy.

1. Evrysdi
People with spinal muscular atrophy cannot produce survival motor neuron (SMN) proteins at sufficient levels due to a mutation in the SMN1 gene. The low levels of this protein cause motor neuron cells to shrink and eventually die. While another gene called SMN2 can also produce SMN proteins, the body can only use a small percentage of the protein. Approved by the U.S. Food and Drug Administration (FDA) in August 2020, Evrysdi is a gene therapy used to treat all types of spinal muscular atrophy. Evrysdi increases the number of functional SMN proteins in the body by targeting the SMN2 gene. The medication is available in liquid form and can be taken orally.

2. Spinraza
Spinraza, developed by Biogen, was the first gene therapy medication approved by the FDA in December 2016. It is still commonly used to treat spinal muscular atrophy in both infants and adults. The medication contains antisense oligonucleotides (ASO) that enhance the cell’s ability to produce more functional SMN protein from the SMN2 gene. Spinraza is administered in the form of an injection. The medication is injected into the fluid around the spinal cord.

3. Zolgensma
Zolgensma is yet another FDA-approved gene therapy used to manage spinal muscular atrophy. The medication works by replacing the function of the missing or impaired SMN1 gene with a healthy copy of the SMN1 gene. FDA approved the medication in 2019 for treating all types of spinal muscular atrophy in infants below 2 years. The medication is administered intravenously as a single dose over 60 minutes. All three types of gene therapy are popular treatment options for people with spinal muscular atrophy.

4. Breathing support
People with spinal muscular atrophy often develop breathing problems due to the weakening of the muscles between the ribs. This can worsen over time as other muscles start deteriorating. If breathing becomes very difficult, patients may likely need ventilation support to get enough oxygen to their bodies. Ventilation can be invasive or non-invasive. In non-invasive ventilation, oxygen is delivered using a mask that covers the mouth. While in invasive ventilation, a tube is inserted down the trachea from the mouth. For long-term ventilation, a tube is surgically inserted through a small incision in the neck.

5. Nutritional treatments
Spinal muscular atrophy can make mouth and throat muscles weak, making it difficult to chew, suck, and swallow foods and liquids. This prevents patients from getting adequate nutrition, resulting in weight loss and several other problems. Some patients also experience acid reflux due to the weakening of the muscles between the esophagus and stomach. While a balanced diet is crucial for managing acid reflux and good nourishment, some people have a tough time eating anything and may require feeding tubes. With the help of feeding tubes, foods can be delivered directly to the stomach.