23-month-old girl with life-threatening disease gets Rs.16 crore drug free of cost

Bhadrachalam: Swiss pharma major Novartis and some Good Samaritans gave a fresh lease of life to Ellen, who is about two years old, at WL Regubaka in Dummugudem mandal, who was suffering from the rare spinal muscular atrophy (SMA).

She was given the 'world's costliest drug’, Zolgensma, which is part of the prescribed gene therapy, that costs Rs. 16 crore free of cost as part of the company’s CSR initiative.

SMA Type 1 is a rare disease that affects one in 10,000 babies worldwide. If not treated immediately, the disease can be life-threatening. The Zolgensma gene therapy is the only treatment option available.

Ellen’s father, Praveen, who came to know that Novartis was giving the injection for free to a country every month, registered her name. On getting it, the injection was administered to the baby in a private hospital in Hyderabad. Praveen said that his daughter is now safe.

Praveen, a medical representative with GSK (Glaxosmithkline) in Eluru, said his daughter was diagnosed with the disease when she was 14 months old. “She was not able to roll and neck-hold due to the disease. She did not have the gene of SMN (Survival Motor Neuron) that produces proteins for neurons to survive, he said.

He tried to mobilize funds through a crowd-funding initiative. A UAE-based Indo-Arabic singer Neha Pandey shared a video on her Instagram seven months back seeking support from her followers for Ellen.

“I am thankful to each and every person who contributed to my child's recovery and, of course, Novartis," said K Ramesh and added that the doctors in Hyderabad who treated her have assured that she will now be getting the neurons and that she would be growing up normally.