Immune Response to Zolgensma Can Be Common in Older SMA Children

Immune Response to Zolgensma Can Be Common in Older SMA Children

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Immune Response to Zolgensma Can Be Common in Older SMA Children

Abnormal increases in liver enzymes levels — suggestive of liver damage — and drops in platelet counts are common among spinal muscular atrophy (SMA) patients over 8 months old following treatment with Zolgensma, according to a small real-world study in Germany. These side effects, which can be severe in some cases, are likely associated with a recognized immune reaction to the modified adeno-associated virus (AAV) used to deliver a working version of SMN1, the mutated gene in…

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