Effective Treatments for Spinal Muscle Atrophy
Spinal muscle atrophy or SMA is a genetic neuromuscular disorder. In this condition, the nerve cells in the brain and the spinal cord break down. Often, SMA affects babies and children, making it difficult for them to move their muscles properly. Although this progressive muscle weakness is rare, it is fatal universally. But, there is no absolute cure for SMA.
The treatment options for spinal muscle atrophy mainly consist of various supportive measures like gene replacement therapy, medications, and other helpful methods. You can learn about those treatment approaches below.
- Nusinersen is a US Food and Drug Administration-approved medication for the treatment of SMA. It is a form of gene replacement therapy. The usage of this medication adjusts the amount of SMN2 genes. By improving the SMN2 gene’s splicing, this medication increases the amount of functional protein required for healthy motor neurons. The FDA has approved the use of this medicine for SMN types 1 to 4. The doctor may inject this medication several times.
- Onasemnogene abeparvovec-xioi is another form of gene therapy approved by the FDA as a treatment option for children less than two years of age with SMA. This gene therapy replaces the defective or missing survival motor neuron (SMN) 1 gene that creates the atrophy with a functional copy. When the infused SMN1 gene reaches the cells in the spinal cord, it increases SMN protein production and improves motor neuron function. Unlike nusinersen, this therapy needs to be done one time only.
- Special breathing support can help patients with SMA types 1 and 2, where the weak muscle makes it difficult to move air in and out of the lungs. A mouthpiece or special mask can make breathing easier. In severe cases, the patient may need machine support for breathing.
- Sometimes, the assistance of a feeding tube may help the babies with SMA get proper nutrition. Children with SMA have weak muscles in the mouth and throat, resulting in difficulties to suck and swallow. Thus, the child may not be getting adequate nutrition. It can also lead to problems related to growth. A feeding tube can help the child avoid such a situation due to SMA.
- When this muscular atrophy starts during childhood, the spine can get a curve. The doctor may recommend that your child wear a back brace during the period of growth to decrease the curvature. In the later period, surgery can rectify the spinal curve.
- For patients with SMA, daily activities and exercise can help maintain a range of motion. Consultation with a physical therapist is advisable for this treatment option.
As SMA is an inherited condition, there is no way to prevent it. Doctors can apply these treatment options for spinal muscle atrophy to manage the symptoms and help them live a quality life. Clinical trials for several therapeutic compounds are on-going and may lead to the emergence of other treatment options.