Spinal muscular atrophy or SMA in children is severe hereditary genetic condition that causes progressive muscle weakness, often resulting in severe disability or death. Gene therapy has emerged as a novel approach to treat SMA in children, aiming at replacing the defective SMN1 gene, addressing the root cause of SMA. To achieve the best possible treatment outcome, a single-dose intravenous infusion of gene therapy is recommended in newborn diagnosed with SMA from after birth until 24 months of age.
Why Gene Therapy at Child’s Health Center, Bangkok Hospital?
- Availability of all pediatric subspecialties
- Team readiness
- Lower treatment cost compared to other countries
👉 For more information, please click: Hope unlocked with GENE THERAPY – A novel treatment for Spinal Muscular Atrophy (SMA) in children
👶 SMA screening in newborns, please click: Newborn Screening Package for Spinal Muscular Atrophy (SMA)
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