Kind donors continue to battle dreaded disease whose remedy costs crores

Afrah Rafeek with her baby brother Muhammed

In a span of six days, numerous good Samaritans from Kerala and across the world came together to raise Rs 18 crore in six days for 18-month-old Muhammed Rafeek from Kerala, who is suffering from Spinal Muscular Atrophy (SMA)-type 3. Muhammed hails from Matool village in Kannur district and needs the imported gene therapy ‘Zolgensma' worth over Rs 16 crore.

The movement to fund the treatment of children suffering from SMA was started by the parents of Teera Kamat from Mumbai. Mihir and Priyanka Kamat, too, had successfully raised money for their daughter's treatment. The government had also exempted the gene therapy of taxes in Teera's case.

Muhammed's elder sister, a Std IX student, Afrah too, suffers from SMA-type 2 and is wheelchair-bound because of the disease. "I want to see my brother walking. I do not want him to be bound to the wheelchair like me," said Afrah, 15. Afrah's second sibling, Anzila, 7, luckily does not suffer from the disease.

Matool panchayat president Farisha Abid

"I have been wheelchair-bound for a few years. I like to play with my siblings but at times, get severe pain in my limbs and back. I aim to become a civil engineer. My family and I are thankful to the people who supported my brother's treatment," Afrah said.

Treatments for SMA

Dr Razeena, co-founder and director of patient empowerment, CureSMA in Kerala, who is closely working with many SMA children, said, "Afrah is suffering from SMA since she was two years old. Her parents could not find a solution for the disease. Today, we have Spinraza, an antisense oligonucleotide intrathecal injection that is given to the spinal cord. It is, however too costly. In 2019, a new drug was developed by Novartis. The gene therapy is a one-time infusion and replaces the SMN1 gene by using a vector AAv9. The infusion is recommended only up to two years of age."

Muhammed's parents realised that he has SMA when he started facing difficulty in walking at the age of one. "They consulted paediatric neurologists at Aster MIMS hospital and were told of the access programme managed by Novartis. Muhammed has only four months left before he loses his eligibility for the treatment," Dr Razeena said.

Muhammed's parents Rafeek and Mareyumma (centre) with community members in their village, Matool in Kerala

"They decided to go for a crowdfunding programme. Afrah's plea for her brother did wonders," Dr Razeena said.

‘Monitored son closely'

Rafeek, employed as an air conditioner (AC) technician in Abu Dhabi for over two decades, said that his mother-in-law, Ummu Saudha, was the biggest support in taking care of the children. She died at the age of 64 a few months ago.

Rafeek added, "When Muhammed started crawling, I would ask my wife, Mareyumma, to monitor his movements closely. During my last vacation, I noticed that he was not climbing stairs nor walking like a normal child his age. Doctors confirmed SMA-type 3. I did not want him to suffer too. We discussed the matter with the village panchayat president Farisha Abid, who formed a committee and made the crowdfunding possible."

"We will always pray for the well-being of each and every donor. We appeal to the governments of Kerala and India to support SMA children. The gene therapy should be made available in India with tax exemptions so that many more children can be saved," said Rafeek, who may return to Abu Dhabi after Muhammed's treatment.

Teamwork gave results

Panchayat president Farisha told mid-day, "I was not sure if I would be able to do much but I decided to support the family in every possible way. We formed a medical committee to help Muhammed. In a month's time, the committee prepared a plan and implemented it. A letter was published in three languages - English, Arabic and Malayalam to reach out to people. A video was made and shared on social media platforms."

"Soon, money poured in from all over the world and the target was achieved in six days. Many NRI organisations from the Arabian Gulf, UA, UK and Europe called us. Muhammed's parents are extremely happy. This was made possible only with teamwork," Farisha said, adding, "I request the government to exempt the taxes on the import of Zolgensma and to give free treatment to children suffering from SMA."