Spinal muscular atrophy causes the nerve cells in the spinal cord and brain to break down, making it difficult for the body to use the muscles properly. The condition is most common among children, and there are only two Food and Drug Administration (FDA)-approved gene therapy treatment options for spinal muscular atrophy. However, this condition can be managed through other methods as well:

• Nusinersen:  This gene therapy is used for both adults and children with spinal muscular atrophy. The treatment is used to modify the SMN2, one of the genes that is influenced by this disease. After the modification, the SMN2 gene can produce more protein. For this treatment, a drug is injected into the spinal cord fluid, and the process takes about 2 hours; and another injection is required after 4 months. This method slows down the progression of the disease and can make the patients stronger. According to studies, it helps around 40% of the patients.
• Onasemnogene abeparvovec-xioi:  This is another effective gene therapy treatment option for spinal muscular atrophy and is used for children younger than 2 years. It is used to replace the defective SMN1 gene to treat the disease. For this treatment procedure, a catheter is inserted into a hand or arm vein to send an SMN gene copy into a targeted motor neuron cell group. This treatment is performed only once, and the therapy helps children with their development milestones, including sitting up and controlling the head.
• Help with breathing:  Apart from treating the disease, its symptoms can also be managed with different methods. For children with spinal muscular atrophy, breathing can particularly be problematic as the weak muscles make it hard for them to breathe easily. This can be managed by wearing a special type of mask or a breathing machine.
• Special feeding methods:  Children with spinal muscular atrophy find it hard to swallow food as the throat muscle and the muscles in the mouth required to swallow the food are rendered weak by this condition. This leads to a lack of nutrition as it is difficult to feed them properly. To tackle this condition, one can work with an experienced nutritionist, and feeding tubes are also used in some cases.
• Physical therapy:  Weak muscles make it difficult for children to walk or maintain natural movements, and physical therapy can help in improving their muscle strength. Other than that, a therapist may also suggest certain methods to aid the child, including using a wheelchair, walker, braces, and computerized tools that can help with drawing and writing.

The treatment options for spinal muscular atrophy are required for managing the symptoms of this condition, and physiotherapists, pulmonologists, neurologists, orthopedists, gastroenterologists, and nutritionists play vital roles.