Treatments that help cope with spinal muscular atrophy
Remedies & Treatments
Spinal muscular atrophy is a neurological condition and a type of motor neuron disease. People with this genetic disorder cannot control their muscle movements because of the loss of nerve cells in the spinal cord and brain stem. While there isn’t any cure or prevention method for the condition, there are treatments to help a person cope with SMA throughout their life.
As SMA treatments are still under the microscope, there are very few medications approved by the Food and Drug Administration (FDA).
- Nusinersen (Spinraza)
This medication helps treat SMA of all types in both children and adults. The doctor will inject four initial doses over approximately two months. It is followed by one dose every four months to maintain the effects of the medication. Nusinersen helps adjust the SMN2 gene and lets it create more protein. While the effectiveness of the medication may vary from person to person, it helps slow SMA progression and even reduces muscle weakness.
- Onasemnogene abeparvovec-xioi (Zolgensma)
This medication helps treat infants below the age of two in the form of an infusion in a single dose. A catheter (tiny tube) is inserted into a vein in the child’s arm or hand. After this, the medical team sends a copy of the SMN gene through a tube that focuses on a specific group of motor neuron cells. Studies show that the medication has helped children affected by SMA achieve developmental milestones quicker. An example is controlling their heads or sitting up without any support.
- Risdiplam (Evrysdi)
It works towards stopping SMN2 genes from disrupting protein production and enabling the protein to reach the nerve cells where required. The dose is given to a child with spinal muscular atrophy once a day following a meal. The size of the dosage is determined based on the weight of the child. It was concluded through clinical trials that Risdiplam helped improve muscle function after 12 months among 41% of patients who consumed it.
Assistive devices and therapy
Other alternative treatments help cope with the condition and increase life expectancy. Assistive technologies such as ventilators, powered wheelchairs, and modified access to computers, help people with SMA become more active and participate in the community. There are physical therapies, too, that include water therapy and wheelchair sports.