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Gillette Children's clinical team recently administered the first dose of Itvisma, a gene replacement therapy for spinal muscular atrophy (SMA). This achievement makes Gillette one of the first hospitals to offer this innovative treatment, highlighting our reputation as a leader in care for children with rare and complex conditions.
Targeting the Root Genetic Cause of Spinal Muscular Atrophy
Itvisma is the intrathecal formulation of Zolgensma, meaning it is delivered directly into the cerebrospinal fluid rather than through an intravenous infusion. Like Zolgensma, it targets the root genetic cause of spinal muscular atrophy by delivering a functional copy of the SMN1 gene, the gene responsible for supporting motor neuron survival and function.
SMA is a devastating neurological disease that, without intervention, progressively weakens a child’s ability to move, breathe, and swallow. For families navigating this diagnosis, access to the latest therapies can be life-changing. By being among the first in the region to offer Itvisma, Gillette Children’s is ensuring that children don't have to travel far from home to access the most advanced treatments available.
Your Support Makes It Possible
Milestones like this one don't happen without investment in people, technology, and the infrastructure that makes complex care possible. Donor funds directly support Gillette Children's continued commitment to advancing care for children with the most challenging conditions. When you give to Gillette, you are helping ensure that the next breakthrough therapy reaches the children who need it, right here in our community.
