Medications
Certain medications have the FDA’s approval and work best for the treatment of symptoms of spinal muscular atrophy. The two medications that are very popular in treating spinal muscular atrophy patients interfere with the human body’s genes and enhance the production of specific proteins to make the muscles stronger. The medications having FDA approval have come under gene therapy, which means that these medications are best for treating newborns and growing children. The two genes involving SMA are SMN1 and SMN2 genes, and the gene therapy helps in giving instructions to these genes, so there is the development of controlled movements of the muscles.
Onasemnogene abeparvovec-xioi:
The use of this specific gene therapy helps in replacing the faulty gene of SMN1 and improves the patient’s condition. Onasemnogene abeparvovec-xioi medication is specifically best and more effective for the children and does not work well for the adults. A team of health care professionals first thoroughly diagnose and monitor children under the age of 2 years and then start the gene therapy. The doctors inject the medication by first inserting the small tube, i.e., catheter into the children vein locating at arm or hand. After direct injection of medication, the doctors insert a weaker copy of SMN genes into the body in the areas with specific motor neuron cells. This type of gene therapy is usually performed once in the life of the children. The use of Onasemnogene abeparvovec-xioi medication dramatically helps the children in controlling their movements like sitting or standing on their own, thus making the children achieve certain developmental milestones faster than the other SMA patients.
Nusinersen:
This gene therapy helps in inducing the production of more muscular proteins by influencing the SMN2 gene. After taking nusinersen medication, there is more protein production in the muscles, which brings controlled movements. This medication is very much useful for both the adults and the babies suffering from spinal muscular atrophy. A health care professional injects the medication into the body of children and adults in the fluid cavity around the spine. The studies on the SMA patient show positive results in the delaying of diseases and make them feel more substantial. The recurrent use of this gene therapy is very helpful for keeping the disease at bay and it does not promise the complete cure for the disease.
Moreover, this therapy’s use is useful in almost 40% of the patients by making their everyday life easier. It is easy to get this gene therapy at a doctor’s clinic and only takes 2 hours to prepare the injection. To get significant results, it is essential to repeat gene therapy several times in four months.