Husna, Amity University Kolkata
United Kingdom’s National Health Service (NHS) has approved the most expensive drug of ₹18 crores (£1.79 million) per dose to cure a rare genetic disorder. The drug Zolgensma, manufactured by Novartis Gene Therapies can treat Spinal Muscular Atrophy (SMA) which is a rare and often fatal genetic disease that causes paralysis and numbness in muscle.
NHS England has moved mountains to make this treatment available since SMA is the leading genetic cause of death among babies and young children, which is why it will be used to treat babies born with severe type 1 SMA disorder which has a life expectancy just two years.
Studies have already shown that this drug Zolgensma is a life changer as it has helped babies suffering from this cruel disease to reach milestones such as breath without a ventilator, sit up on their own and crawl and walk only after a single infusion treatment. The treatment is done by a single intravenous infusion which contains an active ingredient that passes into the nerves, restores a missing gene, and produces the proteins essential for the functioning of the nerve and controlling the muscle movement.
The approval of this drug Zolgensma has marked to be the second medical treatment available for youngsters suffering from SMA, after the drug Spinraza which became available on the NHS to eligible patients in May 2019.
One thought on “Zolgensma: World’s most expensive life-saving drug approved”