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Articles by Novartis

Aniya’s story: how SMN2 backup copy number delayed early treatment

When Aniya was diagnosed with spinal muscular atrophy (SMA), her parents, Hailey and Will, quickly learned that early treatment was essential. It was the only way to stop their baby from losing irreplaceable motor neurons that help the muscles work properly, which would help Aniya avoid disability later in life.

ZOLGENSMA: The One and Only Single-Dose Treatment for SMA

Any parent who has received a spinal muscular atrophy (SMA) diagnosis for their child can tell you that early treatment is crucial. Children who receive treatment for SMA as soon as possible are more likely to achieve important motor milestones. One-time gene therapy ZOLGENSMA® (onasemnogene abeparvovec-xioi), can stop the…