Lucky toddler gets Tk22cr injection shot as ray of hope

Twenty-two month old Raihan, diagnosed with a rare Spinal Muscular Atrophy (SMA), has been unable to walk unlike other children his age, but doctors are now hopeful that a gene therapy – the first of its kind in the country – can change his circumstances.

The treatment for SMA, which is a neurodegenerative disease, is very expensive – costing Tk22 crore. But Raihan will get the medicine free of cost under a global project thanks to the efforts of his doctors at the National Institute of Neurosciences Hospital and a few strokes of luck.

Raihan's gene replacement therapy will begin at Neurosciences hospital at 12 noon today.

Spinal muscular atrophy is a rare and complex congenital disorder of the nervous system, which is caused by genetic factors.

Doctors say children with the condition cannot sit or stand although their neck is not stiff. Due to weaker muscles their movement is limited and they suffer from frequent shortness of breath. The muscles responsible for breathing gradually weaken, resulting in death.

The Neurosciences hospital gets some 30 patients with SMA every year while Shishu Hospital, Bangabandhu Sheikh Mujib Medical University, Dhaka Medical College Hospital also treat children with the condition.

Unlike most SMA patients, Raihan was lucky enough to be selected in a global project lottery, under which multinational pharmaceutical company Novartis provides their gene therapy medication Onasemnogene Abeparvovec to two children from around the world every month for free as part of their CSR campaign.

Doctors hope that Raihan, a Manikganj child, will be able to lead a healthy life after the therapy. Rayhan's father is a Saudi expatriate and his mother a homemaker. 

From Raihan's diagnosis to enrolling his name in the global project, his physician Dr Jobaida Parvin, junior consultant, Paediatric Neurology of Neurosciences hospital hospital, worked to bring the medicine to the country after the lottery.

"Raihan came to the neuroscience hospital when he was seven-eight months old. We initially suspected spinal muscular atrophy, but we do not have the scope of genetic molecular study in our country to confirm the disease," Dr Jobaida told The Business Standard.

"We usually do the Nerve Conduction Velocity test in such patients. After doing that, we saw that his nerves were being damaged and recommended his patients to do a test from India if they can afford it to confirm the diagnosis," she added.

Raihan's family had the test done from India and the doctors confirmed that he was indeed suffering from SMA.

"It was around the same time we learned that a global lottery could provide medicine for this disease. Since 2021, I have been working on sending documents and other procedures to participate in the lottery. We enrolled Raihan's name in April this year and his name came up in the lottery on 14 September," said Dr Jobaida, adding that the medicine came to the country on Sunday.

With the help of Norvatis (Bangladesh) Limited, arrangements have been made to provide this valuable medicine to Raihan.

Dr Jobaida said that the gene replacement therapy medicine will be administered through injection, following which Raihan will be kept under observation for 15 days.  

Raihan is being treated by a 10-member physician team, including paediatric neurology specialists, ICU specialists, nurses and pharmacists.

"All doctors, including the director of our institute, the head of the paediatric neurology department, have been very helpful for Raihan's treatment and the National Board of Revenue waived Tk1 crore tax on the medicine with a short notice," Dr Jobaida said.

"We have very little time as Raihan will be two years old in two months. Since this medicine is not given to children older than two, Raihan can be called lucky. Everything has gone well so far and we are hopeful that Raihan will recover," added Raihan's physician.