Deen Mohammed, a 16-month-old boy from West Bengal, has received a life-changing gene therapy treatment for Spinal Muscular Atrophy (SMA), a rare and debilitating genetic disorder. The treatment was administered at a renowned hospital in Kolkata, utilizing Zolgensma, a groundbreaking gene therapy valued at Rs 16 crore.
Deen's journey to diagnosis and treatment began when his mother, Dilwara Sultana, a daily wage earner, noticed alarming mobility issues in her son at just six months old. Despite facing financial struggles and abandonment by her husband, Dilwara sought medical advice, driven by her determination to help her child.
Initial consultations with local pediatricians led to a recommendation for treatment at Kolkata's Peerless Hospital. However, the financial burden seemed insurmountable. Fortunately, Dilwara's brother, a farmer, provided crucial financial support, enabling the family to pursue treatment.
At Peerless Hospital, Deen was diagnosed with Spinal Muscular Atrophy (SMA), a complex and challenging disease requiring expensive treatment. SMA affects infants motor neurons and nerve cells, impairing voluntary muscle movement. The disorder is rare, with a worldwide prevalence estimated at 1 in 8,000.
In a remarkable turn of events, Deen's name was selected through a lottery system for free treatment under a specialized program at Peerless Hospital. This global trial, offered by a US-based pharmaceutical company in 36 countries, concluded on July 31.
The hospital's commitment to treating SMA, citing its comprehensive ecosystem and dedicated doctors. We are dedicated to delivering exceptional care to children with SMA.
Ravindra Pai, Managing Director of Peerless Hospital
Ravindra Pai, Managing Director of Peerless Hospital, emphasized the hospital's commitment to treating SMA, citing its comprehensive ecosystem and dedicated doctors. We are dedicated to delivering exceptional care to children with SMA," Pai emphasized.
Following administration of the life-saving medicine, Zolgensma, Deen was placed in isolation and is expected to recover soon. After discharge, he will undergo rigorous physiotherapy and regular follow-ups. Doctors anticipate significant improvement in limb movement within two years.
Sanjukta Dey, trained to administer the medicine, highlighted the importance of follow-up visits and shared insightful data on SMA's prevalence. "The carrier rate is 1 in 35, making it the second-most common genetic disorder after Thalassaemia," Dey noted.
"The carrier rate is 1 in 35, making it the second-most common genetic disorder after Thalassaemia," Sanjukta Dey noted.
Ravindra Pai also advocated for collaborative efforts to combat SMA, stressing the need for private-public partnerships, increased funding initiatives, and robust national policies for rare diseases.
Deen's treatment marks a beacon of hope for Indian families affected by SMA, who often find treatment unaffordable due to exorbitant costs. Crowdfunding efforts are underway to support more SMA patients, with three children (7-15 months) awaiting funding for treatment, which is most effective before age two.
(Input from various sources)
(Rehash/Sai Sindhuja K/MSM)
