Spinal muscular atrophy treatment with gene replacement therapy

Spinal muscular atrophy treatment with gene replacement therapy remains among the newest yet most promising methods that can help prevent any symptoms if started early and performed consistently. Nowadays, many techniques that can help replace a malfunctioning gene causing spinal muscular atrophy with a normal one undergo clinical trials, and few are already being approved. Right before the start of treatment, medical practitioners need to get as much information about the patient's health and disease as possible, so they prescribe various diagnostic procedures and lab tests. Onasemnogene abeparvovec-xioi (Zolgensma) helps to replace the malfunctioning SM1 gene with a normal one and uses the so-called scAAV9 delivery approach. In this drug, adequately functioning SM1 genes are associated with adenovirus that enters the nerve cells and helps to integrate normal genes into the nerve cells. As a result, the patient's motor neurons start to function correctly. Also, the treatment leads to the production of healthy nerve cells in the future. Zolgensma can be delivered via intravenous injections, injections in the spinal canal, or other methods, depending on the patient's age, current condition, and severity of symptoms. Gene replacement therapy is one of the best methods for kids, yet getting a neurologist's appointment is essential to ensure they can successfully enroll.