Mattool child given SMA drug | Kozhikode News – Times of India

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Kozhikode: The Spinal Muscular Atrophy (SMA) treatment of one-and-a-half-year old Mohammed from Mattool in Kannur, for whom Malayalis had shown unprecedented benevolence by donating Rs 46 crore, began on Tuesday with the administration of Zolgensma drug which costs almost Rs 18 crore.
The gene therapy drug was administered at Aster MIMS hospital under the supervision of paediatric neurologist Dr Smilu Mohanlal. Hospital authorities said that Mohammed will have to remain at the hospital under observation for a few days. The kid was diagnosed with SMA type 2 and 3, a rare genetic disease that weakens the muscles and leads to walking difficulty, after early childhood. The child had to be given the gene therapy medication before he reached the age of two.
The medicine targets the genetic root cause of SMA by replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions.
Central government had waived duty for the import of 74.2ml dose of Zolgensma drug for Mohammed’s treatment
The family had hoped to collect Rs 18 crore through crowdfunding for the gene therapy treatment; but Rs 46.7 crore was contributed by the people. It was decided to hand over the excess amount to the treatment of other SMA-affected children.

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