10-month-old baby boy becomes first in Hong Kong to receive life-extending gene therapy treatment for rare neuromuscular disorder
- The Hospital Authority has introduced a single injection of a new drug called ‘Zolgensma’ for Spinal Muscular Atrophy (SMA) patients
- ‘With this new treatment, patients only need a single injection and do not need to be hospitalised regularly. I believe this is very helpful for patients and their families,’ expert says
A 10-month-old baby with a rare neuromuscular disorder has become the first in Hong Kong to receive a single-use gene therapy treatment for the deadly disease, with doctors saying it can increase the life expectancy of patients.
The Hospital Authority on Saturday said that it had introduced a new type of gene therapy using a single injection of a new drug called “Zolgensma” for Spinal Muscular Atrophy (SMA) patients.
The genetic disease, which one in every 10,000 babies is born with, affects the nerve cells controlling the muscles.
The new drug will reduce the burden of the disease among patients and their families, according to the authority.
“The current treatments include an injection to the spine that needs to be received every three to four months in a hospital,” Simon Tang Yiu-hang, the authority’s director of cluster services, said. “Another treatment is a daily tablet.