In what can be described as a humanitarian gesture, a 23-month-old baby named Ellen, was given Zolgensma Gene Therapy injection which costs Rs.16 crore free of cost in Rainbow Children’s Hospital in Hyderabad.

Ellen was suffering from Spinal Muscular Atrophy-1 (SMA-1) – a rare genetic disorder – for which this injection is the only treatment. Her parents, Rayapudi Praveen and Stella, who are residents of Reguballi village in Bhadradri Kothagudem district in Andhra Pradesh had put out an appeal to raise funds for this injection last year.

The Switzerland-based pharma company Novartis, provided the drug free of cost for Ellen.

Talking to the media about this disorder, Dr Ramesh Konanki who is a Consultant Paediatric Neurologist, informed that the children affected by SMA-1 are unable to attain growth milestones.

Ellen was unable to control her voluntary movements including the motion in neck, hands, head and legs. She couldn’t even swallow. Besides, she had breathing difficulties and suffered from extreme weakness in muscle because of which she couldn’t hold her neck or sit.

Sharing more details, Dr. Ramesh said: “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.”