The therapy has been found to be effective with the proper production of SMN proteins.
New gene therapy has been approved by the U.S. Food and Drug Administration that could save the lives of babies with a rare disorder, spinal muscular atrophy (SMA). However, the therapy is likely to cost around US$ 2.1 million, priced by the pharmaceutical company Novartis, making it the most expensive drug in the world to date. The gene therapy is called Zolgensma is for children under age 2 years with SMA, including those who have not shown symptoms. The drug will free the babies from one of the deadliest diseases as well as with types where debilitating symptoms may set in later.
Dr. Emmanuelle Tiongson, a pediatric neurologist at Children’s Hospital “This is potentially a new standard of care for babies with the most serious form of SMA. The job now is trying to negotiate with insurers that this would be long-term savings.” Zolgensma was developed to target a rare and inherited genetic mutation in the SMN1 gene. Children born with the mutation are unable to produce effective volumes of SMN protein, which is extremely vital for motor neurons in the spinal cord and brain. Moreover, in its extreme case, it leads to death in the majority of children.
In this therapy, a copy of the SMN gene is delivered directly into motor neuron cells. The therapy has been found to be effective with the proper production of SMN proteins. Peter Marks, working at FDA’ Center for Biologics Evaluation and Research said, "Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure." "Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival," he added further. Although the therapy is effective, it crucial how many parents will be able to it.
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