HYDERABAD/KHAMMAM: A 23-month-old baby suffering from Spinal Muscular Atrophy-1 (SMA-1), a rare genetic disorder, was administered Zolgensma Gene Therapy, the only treatment available, in the form of an injection that costs Rs 16 crore, at the Rainbow Children’s Hospital in Hyderabad on Sunday.
Novartis, the Switzerland-based pharmaceutical firm, supplied the drug-free of cost for Ellen, daughter of Rayapudi Praveen and Stella, residents of Reguballi village in Bhadradri Kothagudem district.
Dr Ramesh Konanki, the consultant paediatric neurologist, said that children affected by SMA-1 are unable to attain growth milestones.
Ellen couldn’t control her voluntary movements, especially motion in the head, neck, hands, and legs, and was unable to swallow. She also developed breathing difficulty and severe muscle weakness due to which she couldn’t even sit or hold her neck.
“SMA can be life-threatening, if not treated before the child attains two years of age. Until 2019, there was no treatment. However, Novartis developed a treatment that works by replacing the defective gene,” Dr Ramesh said.
This was the fourth case of the drug being administered at the hospital.