A 16-month-old child diagnosed with spinal muscular atrophy (SMA) has received a potentially life-altering gene therapy at a private hospital in Kolkata. The infant, Deen Mohammad, whose father is a daily wage earner from East Midnapore, was selected to receive Zolgensma, a drug valued at approximately ₹17.5 crore, free of charge as part of a global trial.
Details of the Treatment
Deen received the gene therapy on Monday and was subsequently placed in an isolation ward for observation. Doctors anticipate discharging him on Wednesday, after which he will begin a rigorous physiotherapy program and attend regular follow-up appointments. According to medical staff, it may take up to two years for Deen to regain full movement in his limbs.
Background on SMA
SMA is a rare genetic disorder that affects motor neurons and nerve cells, leading to muscle weakness and atrophy. It is considered the second most common genetic disorder after thalassemia, with a carrier rate of 1 in 35, according to recent data. While currently incurable, its symptoms can be managed through various therapies.
Deen's mother, Dilwara Sultana, noticed his inability to move his limbs when he was six months old. "I took my son to a local doctor who advised me to take him to a hospital in Kolkata... Those at the hospital enlisted my son for a programme, and by lottery, his name was chosen for the free treatment," said Dilwara.
Calls for Increased Access and Funding
Sanjukta Dey, who is specially trained to administer the medicine, highlighted the importance of follow-up care. Ravindra Pai, MD of Peerless Hospital, emphasized the need for public-private partnerships to facilitate early detection and treatment, along with increased funding initiatives under the national policy for rare diseases.
Jasodhara Chaudhuri, assistant professor of neuromedicine at NRS, mentioned that the US-based pharma company has applied for permission to sell the drug in India. She urged the government to expedite the approval process to improve access for families and to invest more in gene therapy research. "Effort is underway to fund more SMA patients through crowdfunding initiatives. We urge the govt to quickly approve the medication so that children from ordinary families can access it and to focus more on research in gene therapy."
