By Dr Nicola Davies
Adenosine deaminase (ADA) deficiency is an inherited systemic metabolic disorder that damages the immune system and causes severe combined immunodeficiency (SCID). It occurs in approximately one in every 200,000 to 1,000,000 newborns worldwide.1 We take a deep dive into the current treatment options, treatment gaps, and future directions pharma can take to improve treatment for this rare yet serious condition.
What is ADA deficiency?
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