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Is my child a candidate for Gene Therapy?​

Your child’s doctor will decide if your child is eligible to get the gene therapy​

Inclusion Criteria:​
  • Your child should have a confirmed genetic diagnosis of SMA​
  • The age is less than two years OR has a weight less than 13.5 kg ​
  • All baseline bloods are normal​
  • Negative AAV-9 antibody test​
Exclusion Criteria:​
  • A child on the ventilator​
  • A child with a tracheostomy + any form of ventilation (BiPAP, CPAP)​
  • Weight > 20kg​
  • A child with a long term cardiac, liver, brain or kidney condition​

Al Jalila Children's Specialty Hospital

Al Jaddaf - Dubai United Arab Emirates

800 AJCH (8002524)
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