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Medcare aids two infants with gene therapy worth $2.1m

Dubai - Medcare Hospitals has become the first healthcare provider in the Middle East to offer the complimentary gene therapy to two infants suffering from spinal muscular atrophy (SMA) type 1 as part of the managed access programme (MAP) by Novartis.

Novartis, a pharmaceutical company that develops this gene therapy for SMA, started this programme in 2020 to offer gene therapy for free to 100 patients in the world diagnosed with SMA. An infant is selected every 15 days as the part of this programme, and the first two in the region have been chosen from Medcare Women & Children Hospital (MWCH).

SMA is a genetic condition in which an essential protein called SMN is not produced as a gene responsible for its production called SMN1 is deleted or mutated, making the muscles weaker. The condition worsens over time and can be fatal. A severe case (type 1) can mean that the child may not survive beyond a couple of years. However, with gene therapy, the patient can lead a normal, or close to normal life.

Dr Vivek Mundada, a paediatric neurologist at MWCH, said: "Gene replacement therapy means replacing with a fully functional copy of the missing SMN1 gene, which is the root cause of the disease. The gene then enters targeted body cells and starts producing the missing SMN protein."

"Children with SMA Type 1, who usually never achieve the developmental milestone of sitting and have received this gene replacement therapy, have learned to sit and have survived with good quality of life," Dr Mundada said.

Both the children receiving gene therapy suffer from SMA type 1 and will be the first in the Middle East to receive gene therapy under the MAP programme. Apart from Dr Mundada, they have been seen by paediatric respiratory and gastrointestinal specialists.