Indication

Adenosine deaminase severe combined immune deficiency

Adenosine deaminase severe combined immune deficiency is an autosomal recessive inborn error of metabolism and primary immunodeficiency caused by mutations in the *ADA* gene. Read more about Adenosine deaminase severe combined immune deficiency

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Common Adenosine deaminase severe combined immune deficiency therapies.

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REVCOVI (elapegademase-lvlr)

METABOLIC DISEASE ENZYME REPLACEMENT, SEVERE COMB IMMUNE DEF·ADENOSINE DEAMINASE SEVERE COMBINED IMMUNE DEFICIENCY
CHIESI USA, INC
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About Adenosine deaminase severe combined immune deficiency.

Adenosine deaminase severe combined immune deficiency is an autosomal recessive inborn error of metabolism and primary immunodeficiency caused by mutations in the *ADA* gene.

The deficiency of the adenosine deaminase enzyme causes intracellular accumulation of toxic purine metabolites, primarily deoxyadenosine and deoxyadenosine triphosphate, which trigger premature apoptosis of rapidly dividing lymphocytes.

This leads to profound, combined T-, B-, and natural killer-cell lymphopenia and secondary systemic abnormalities.

Primarily affecting infants within the first six months of life, the condition is clinically characterized by severe failure to thrive, persistent diarrhea, and life-threatening, recurrent opportunistic infections.

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