HA introduces gene therapy for spinal muscular atrophy
The Hospital Authority (HA) on Saturday said it has introduced a gene therapy injection for infants suffering from spinal muscular atrophy (SMA), and that it is more effective than traditional treatments for the illness. It said a 10-month-old infant was the first patient in Hong Kong to receive this therapy, called onasemnogene abeparvovec. Dr Simon Tang, the HA's cluster services director, said a patient's survival rate can increase after just one injection, and the therapy is at its most effective when used on patients under the age of one. "There were originally two kinds of medication helping SMA patients. One of them requires intrathecal injection, and patients normally need to go back to the hospital for injection every three to four months. The other medication is an oral drug, but patients have to take the drug everyday," he said. "The advantage of this gene therapy is [that it’s] a one-time treatment. Basically, patients just have to do one injection, instead of going back to the hospital for regular injections or taking drugs everyday. This can minimise the inconvenience of long-term treatments." Dr Tang added that there can be up to five infants in Hong Kong annually who suffer from SMA, and that they would be suitable candidates for the new therapy. The HA added that though the new therapy is not subsidised, families can apply for the treatment under a medical assistance programme for those in need.