The disease is treated either with a drug, called Sprinraza, that is administered every four months, or with a gene therapy, called Zolgensma, which is a one-off cure for babies under the age of 2 or below 21kg. After two weeks, she will not be eligible to apply for gene therapy, which is available in Hungary. “Babies who use the first drug carry the risk of death, while the gene therapy is successful with 95 percent of patients. Although Koca said the state already covers the expenses of SMA patients, not all children can benefit from Sprinraza-based treatment. According to Yarli, families want to apply the one-off gene therapy before the age of 2 because its effectiveness has been proved globally.

Source: Ethiopian News January 04, 2021 23:15 UTC