Drug treats SMA(spinal muscular atrophy) a rare n often fatal genetic disease which will be used to treat babies and young children.
Babies born with SMA have a life span of around 2years.
Zolgensma, has helped babies to reach milestones such as breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment, showed studies.
The treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1. The active ingredient onasemnogene abeparvovec passes into the nerves and restores the gene, which then produces proteins essential for nerve function and controlling muscle movement, it explained.
