Spinal muscular atrophy (SMA) is a neurodegenerative condition. It is characterized by the progressive weakening of muscles in the body. The condition arises due to the patient’s inability to produce the survival motor neuron. Although there is no cure for the disease as yet, patients can try out a combination of treatment options in order to manage the condition better.

Medications

• Nusinersen
  Marketed as Spinranza, nusinersen is the first Food and Drug Administration (FDA)-approved oral medication for spinal muscular atrophy patients. The drug is said to make alterations in the SMA2 gene, allowing it to produce more proteins. The drug belongs to the class antisense oligonucleotides (ASO). This class of drugs influences RNA production and is aimed at targeting the underlying problem. Nurisergen has shown results in helping slow down the progression of SMA decreasing muscle weakness. The medicine is administered directly into the spinal fluid and has to be given every few months.
• Zolgensma
  Zolgensma is a gene therapy medication that employs the use of a genetically engineered virus. This virus helps treat SMA by delivering a healthy SMN1 gene copy to the body, resulting in a higher level of functional proteins. Zolgensma is a fairly new entrant from the various treatment options for spinal muscular atrophy, and can only be used in patients of up to two years of age.

Breathing assistance

Spinal muscular atrophy has been known to weaken the muscles that lie between the ribs, leading to difficulty in the expansion of the chest while breathing. This proves it difficult for the lungs to function normally and puts excessive pressure on the diaphragm. This weakness aggravates with the progression of the condition. Due to this, patients may require breathing assistance by means of ventilation. The kind of assistance that has to be administered is decided after examining the symptoms of the patient. Short-term assistance is provided by means of non-invasive ventilation, wherein the patient is provided with oxygen through a mask. In the case of long-term assistance, invasive ventilation is administered by inserting a tube down the patient’s trachea. The tube is passed down the patient’s mouth or is inserted surgically via an incision in the patient’s neck.

Occupational therapy

Occupational therapy, as a treatment option for spinal muscular atrophy, involves the use of assistive instruments to help the patient in maintaining a fairly independent lifestyle and regular schedules. These devices may include, but are not limited to, braces, wheelchairs, and speech synthesizers.

Physical therapy

Another possible treatment option for spinal muscular atrophy includes physical therapy. It helps maintain the patient’s range of motion and muscle strength for as long as possible. However, in SMA patients, excessive exercise can lead to problems. It is, therefore, advisable for patients to seek professional guidance before settling for an exercise routine. Seeking advice from a physical therapist can help put together a more suitable routine, and also be beneficial in tracking progress.