Doctors use most expensive drug worth £1.795m to save 5 months old baby

A pack of Zolgensma worth N 1, 044, 546,400

LONDON, UK – Five months old baby, Arthur Reece Morgan will now pass as the first baby to be treated with the world most expensive drug, Zolgensma, which sells for £1.795 million(about N1, 044, 546,400) in the United Kingdom.

Author Reece Morgan is being treated with the drug for Spinal Muscular Atrophy, a disorder that causes loss of ability to control the muscle. If not treated, the disorder could lead to death. Besides, doctors say the drug is not a cure but will only help to manage the patient and prolong his life.

One million seven hundred and ninety-five million British Pounds (£1.795) million is far above what most parents can afford in the UK.

Consequently the NHS has offered to pick up the bills. The BBC reports about 40 children are born with Spinal Muscular Atrophy every year globally. The drug, Zolgensma, manufactured by Novartis Gene Therapies comes in a small pack and it nothing but a healthy copy of the faulty gene, called SMN1.

Writing on the disorder, the NHS, UK said, It’s not currently possible to cure spinal muscular atrophy (SMA), but that research is ongoing to find new treatments.

It added that treatment and support is available to manage the symptoms and help people with the condition have the best possible quality of life. “A team of different healthcare professionals will be involved in you or your child’s care. They’ll help make a care plan outlining the support and treatments you may need”.

Still on the disorder, the National Institute of Health said the most common form of SMA is caused by defects in both copies of the survival motor neuron 1 gene (SMN1) on chromosome 5q.

“This gene produces the survival motor neuron (SMN) protein which maintains the health and normal function of motor neurons. Individuals with SMA have insufficient levels of the SMN protein, which leads to loss of motor neurons in the spinal cord, producing weakness and wasting of the skeletal muscles. This weakness is often more severe in the trunk and upper leg and arm muscles than in muscles of the hands and feet.

“There are many types of spinal muscular atrophy that are caused by changes in the same genes. Less common forms of SMA are caused by mutations in other genes including the VAPB gene located on chromosome 20, the DYNC1H1 gene on chromosome 14, the BICD2 gene on chromosome 9, and the UBA1 gene on the X chromosome. The types differ in age of onset and severity of muscle weakness; however, there is overlap between the types”, the NIH.

It however advised that people with SMA, especially children should get the right nutrients, adding that it will help with healthy growth and development.