Warning of TMA, Rare Blood Disorder, Added to Zolgensma’s Safety Label

Warning of TMA, Rare Blood Disorder, Added to Zolgensma’s Safety Label

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Warning of TMA, Rare Blood Disorder, Added to Zolgensma’s Safety Label

Novartis Gene Therapies has added thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — to the list of safety concerns with use of Zolgensma in children with spinal muscular atrophy (SMA). This decision was driven by reports of six confirmed cases of TMA within one to two weeks of the gene therapy’s single dose being given to children, 4 months to 4.5 years old, between October 2019 and February 2021, a company community announcement stated. Three of…

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