A Baby’s Milestones and the Next Steps for Novartis in Gene Therapy

via Cameron Hewitt’s family celebrates the anniversary of her life not once, but twice yearly: Besides her September birthday, they commemorate a day in March—the anniversary of two-year-old Cameron receiving the gene therapy that gave her a new shot at life.

Cameron was born with spinal muscular atrophy (SMA), a genetic disorder that leads to the progressive loss of muscle function. In its most common and severe form, the disease leads to death within one or two years. The Hewitt family, which lives in Apex, NC, credits Zolgensma, a gene therapy approved by the FDA last year, as the reason Cameron lived to her second birthday and continues to improve today.

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