Telangana baby with rare genetic disease administered Rs 16 crore drug

Hyderabad: A 23-month-old baby with Spinal Muscular Atrophy (SMA) Type 1, received a new lease of life with Swiss drugmaker Novartis coming forward to donate gene therapy costing Rs 16 crore.

Baby Ellen was administered Zolgensma gene therapy, called the world's costliest drug, at a private hospital in Hyderabad on August 6, her father Rayapudi Praveen said on Sunday.

The baby with a rare genetic disease is currently under medical observation.

Hailing from Bhadradri Kothagudem district of Telangana, Praveen and his wife Stella, were struggling to raise the huge money required for the only treatment option for their child.

According to Praveen, they had registered their daughter under Managed Access Programme of Novartis and they received the information that the company had selected her as the beneficiary.

SMA Type 1 is a rare genetic disease that attacks the baby's nerves and muscles and makes it extremely difficult for the child to carry out basic activities like sitting up, lifting head, swallowing milk, and even breathing.

SMA is currently the leading genetic cause of infant death worldwide, and it affects one in 10,000 babies.

Praveen, a resident of Bhadrachalam, who works as a medical representative, along with his wife had set up a fundraiser on Milaap to raise the required amount for Zolgensma.

UAE-based Indo-Arabic singer Neha Pandey had also used her Instagram handle to further amplify this cause. However, only Rs 79.36 lakh could be raised.

Zolgensma, made by Novartis, is available only in the US and Europe.

Under the Managed Access Programme, Novartis helps patients with serious or life-threatening diseases or conditions seeking medical products that are not yet approved or available in their country. It makes certain investigational or unapproved treatments available to eligible patients.