Medications
FDA-approved medications are the best to treat symptoms of spinal muscular atrophies. Two medications used to treat spinal muscular atrophy are interfering with genes in the body and enhancing the production of proteins. FDA-approved medications fall under the category of gene therapy. This means they are most suitable for newborns and children who are growing. Gene therapy can help control the movement of muscles by giving instructions to the SMN1 or SMN2 genes.
Onasemnogene abeparvovec-xioi:
This specific gene therapy replaces the faulty SMN1 gene and improves patient condition. The abeparvovec xioi medicine is more effective and best for children. It does not work as well for adults. The team of medical professionals must first diagnose and monitor the children below the age of two years before starting the gene therapy. Doctors inject the medication into a vein located at the arm or hand of the child by inserting a small tube (catheter). The doctors inject a weaker version of the SMN gene into the body after direct injection. This is done in areas where there are specific motor neuron cell. The children usually only undergo this type of gene therapy once. Onasemnogene Abeparvovec – xioi helps children control their movements, such as sitting or standing up on their own. This allows them to achieve developmental milestones quicker than other SMA patients.
Nusinersen:
The SMN2 gene is manipulated by this gene therapy to increase the production of muscle proteins. Nusinersen increases the production of muscle proteins, which results in controlled movements. Both adults and babies with spinal muscular atrophy can benefit from this medication. The medication is injected by a health care professional into the fluid around the spine of both children and adults. Studies on SMA patients show positive results for delaying diseases and making them feel more substantial. This gene therapy can be used repeatedly to keep the disease at bay, but it doesn’t promise a complete cure.
This therapy is also useful for almost 40% of patients, as it makes their daily life easier. This gene therapy is easily obtained at a doctor’s clinic, and the preparation of the injection only takes two hours. For significant results, you must repeat the gene therapy multiple times over a period of four months.
