Our daughter is sinking and we are helpless: Parents of baby with genetic disorder

Baby Teera, hours before she was put on ventilator

How many infants will lose their battle to SMA? How long will it take to bring the therapy to India? Why is the government not listening to our plea? Our daughter is sinking, and we are helpless," ask baby Teera's parents, who have been outside the ICU of SRCC Hospital where the five-month-old is on ventilator support for over 48 hours since her lungs collapsed.

This Valentine's Day Teera will turn six months old, and till date, the family has managed to collect Rs 12 crore out of the Rs 16 crore required for the gene therapy to treat Spinal Muscular Atrophy (SMA) that she is suffering from.

Mid-day had in its articles titled, ‘Baby with genetic disorder needs Rs 16 crore therapy to live,' on December 11, 2020, and, ‘Parents of baby suffering from rare disorder seek help from Maharashtra government,' on December 21, 2020, highlighted the issue. Only government intervention to get the therapy to India at an affordable cost at the earliest can save Teera and other SMA infants. The single shot of gene therapy that can save these children, costs Rs 16 crore.

Baby Teera

Speaking to mid-day, Mihir Kamat, Teera's father said, "On January 13 at about 8.30 pm Teera had a severe aspiration episode and turned blue. She almost stopped breathing. We patted her back, suctioned her airway till she could breathe and showed some sign of normalcy. We then rushed her to the Holy Spirit Hospital emergency room to stablise her condition."

On Thursday morning, Teera had to be taken to SRCC children's hospital in Mahalakshmi through a cardiac ambulance. She was given external oxygen support. Her lungs collapsed and she was put on a full-time ventilator.

‘Waive off exorbitant taxes'

"While various domestic and international crowdfunding platforms helped us collect Rs 12 crore in such a short span, we still need Rs 4 crore. But collecting the additional import tax and GST for imported medicines adds an additional 35 percent to the actual expense of therapy. The government should hence waive off such exorbitant taxes on life-saving imported medicines/therapy and make the therapy affordable for common people in India," said Mihir.

Baby Teera is on ventilator support

Mihir added that they wanted to take Teera to Nationwide Children Hospital, Ohio, USA where the clinical trial for gene therapy Zolgensma for SMA happened. The hospital had even quoted a price of $2.32 million towards the treatment but their plans came to a standstill as Teera is on ventilator support.

‘Cure caught up in red tape'

Alpana Sharma, co-founder and director, patient advocacy, CureSMA Foundation of India said, "SMA's cure is not reaching our children in India, due to red tapism, and only the government's proactive measures will help lift this barrier. It is our humble request to the government to look into the matter to help save our children."
She said the government formed a National Rare Disease Policy but did not address the funding mechanism.

Another SMA child struggles

Ruvanthika and her brother

Ruvanthika, 4, from Tamilnadu, has SMA type 2 and is on a non-invasive ventilator in hospital since January 15. Her younger brother, Kavipriyan, is also SMA type 2 and their parents - Selva Priya and Rajendran - are fighting all odds to save her, said Alpana Sharma.

‘Government should intervene before it's too late'

Alpana Sharma, Co-Founder and Director Patient Advocacy, CureSMA Foundation of India said, "Whose child will it be next? Is a fear that many SMA parents are living with today, with no ray of hope coming in form of support from the state or central government. SMA has a cure, but the cure is not reaching our children in India, due to red tapism, and only government proactive measures will help in lifting this barrier and can bring a ray of hope for SMA children at an affordable price."

"Spinal Muscular Atrophy, a life threatening neuromuscular genetic disease, which did not have any treatments all these years has now got newer lifesaving medicines approved by US FDA in recent times, however, these are not accessible and affordable to Indian patients suffering from SMA," said Sharma.

She added, "CureSMA Foundation of India is committed to Indian SMA Community and it has been our endeavour to bring SMA treatment to India. But the cost of these medicines is the barrier to access and we need assistance from the Government to save SMA patients. It is our humble request to kindly look into the matter to provide immediate intervention through financial support to help save the life of our dying children."

"The Government of India made a National Rare Disease Policy but did not address the funding mechanism which is the root cause of all the problems. The parents and patients of CureSMA Foundation of India have made several attempts and will continue to do so to let government, both at central and state to know that Spinal Muscular Atrophy does not give us luxury of time and that we are losing children and patients rapidly despite a life saving medicine available on this planet," said the Co-Founder of CureSWA Foundation.

"This disease is like a poison slowly spreading in our children's bodies. It's absolutely heart-breaking for us to see our children wasting away in front of our eyes despite the fact that there is a promising medicine available; however just because of lack of funds the lifesaving medicine is not accessible to our children. It's our humble plea to the government to help us save precious life because with progressive and debilitating conditions like SMA it's a Race Against Time," she said.

"No parent should have to undergo the grief of burying their child especially when there is a medicine out there that can save their precious life. We the unfortunate parents and patients of CureSMA Foundation of India live in constant fear, whose turn next? It's high time that the government intervenes immediately before we lose more lives due to this draconian disease called Spinal Muscular Atrophy," she added.