(Reuters) -U.S. Food and Drug Administration has approved Novartis’ gene therapy for a type of rare muscle disorder, the drugmaker said on Monday.
The therapy, branded as Itvisma, was approved for the treatment of spinal muscular atrophy patients of age two years and older who have a confirmed mutation in the survival motor neuron 1 gene.
It addresses the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight, the company said.
Spinal muscular atrophy is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene, which is responsible for the production of a protein needed for muscle function, including breathing, swallowing and basic movement.
(Reporting by Sriparna Roy in Bengaluru; Editing by Leroy Leo)
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