Two Medical Breakthroughs for Spinal Muscular Atrophy Treatment
Dr. Byrne's study
From this, Dr. Byrne and his team developed a new plan. They began studying about gene therapy in 2011. They created three injections of the enzyme that can compensate for ERT into the diaphragm, then into the cells. Out of 9 patients, 4 patients have shown progression and did not need to be on mechanical ventilators.
The gene therapy has wide-ranging results, so it cannot always be a cure. Some medications like Luxturna, which is used to regain the sense of sight, are still needed. Other forms of this type of therapy can cure a disease and prevent symptoms from worsening and appearing that can make a child do other therapies needed.
