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Panel

• Moderator: Dr Andrew Gennery
  Newcastle University and Great North Children’s Hospital, Newcastle upon Tyne, UK
• Dr Claire Booth
  UCL Great Ormond Street Institute of Child Health, London, UK
• Dr Maria Pia Cicalese
  San Raffaele Scientific Institute, Milan, Italy

Summary

Improve your knowledge of the diagnosis and management of ADA-SCID in every day clinical practice. In the context of patient cases, our expert panel discuss topics including the screening and detection of ADA-SCID, the current care pathway and the increasing role novel gene therapies look to play in the treatment of patients with this rare immune disorder.

Learning objectives

At the end of the webcast, participants will be able to:

• Explain the underlying biology as well as the screening and detection methods for ADA-SCID.
• Describe and debate the current treatment algorithm for ADA deficiency.
• Summarise the latest advances in gene therapy for ADA-SCID, notably gammaretroviral and lentiviral vector gene therapies, and how these novel treatments can be applied to every day clinical practice.

Evaluation

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