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Treatment

What would the process of giving gene therapy entail?​

  • Prior to the initiation of gene therapy, we will carry a few tests to help us decide if the gene therapy is suitable for your child. Once these results are normal, the gene therapy treatment will be ordered ​
  • The gene therapy should arrive within 12-14 working days ​
  • One day before the infusion, your child will start steroids​
  • On the day of the infusion your child will be admitted to the daycare​
  • The infusion takes one hour ​
  • After the gene therapy, the child will be admitted to the ward for 24 hours observation or to stay at least for 4 hours after the infusion if you prefer to go home​ 

What are the possible side effects of gene therapy?​

  • First 3 days: Fever and vomiting​
  • Very common: increase in Liver enzymes, decrease in red blood cells and platelet counts, abnormal clotting results ​
  • Rare: Some reports that the gene therapy affects the heart and the kidneys ​
  • The use of steroids is mainly to reduce those side effects​

What is the short-term care after gene therapy?​

  • Your child will be seen weekly for follow up appointments in our neurology outpatient department where we will be conducting blood tests and monitoring their results.​
  • Blood tests done include Liver function tests, Renal function tests, Full blood count, Cardiac enzymes and clotting profile.​
  • These tests will be carried out regularly over a period of at least three months.​
  • The dose of steroids will be tapered according to the blood results​

Al Jalila Children's Specialty Hospital

Al Jaddaf - Dubai United Arab Emirates

800 AJCH (8002524)

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