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ZOLGENSMA is a gene replacement therapy that delivers a working SMN gene to treat spinal muscular atrophy (SMA), a rare genetic disease affecting motor nerve cells. The treatment is designed for infants and children with SMA to help restore survival motor neuron protein production.
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Frequently asked questions
- What does Zolgensma do?
- ZOLGENSMA is a gene replacement therapy that delivers a working SMN gene to treat spinal muscular atrophy (SMA), a rare genetic disease affecting motor nerve cells. The treatment is designed for infants and children with SMA to help restore survival motor neuron protein production.
- Who are Zolgensma's investors?
- Zolgensma's investors include HEALTHCARE ROYALTY PARTNERS.