At the end of May, Zolgensma was approved by the US Food and Drug Administration (FDA) as a gene therapy for children up to the age of two with spinal muscular atrophy (SMA). This was followed on June 3 by the European Commission’s marketing authorization for Zynteglo, a gene therapy against transfusiondependent β-thalassemia in patients 12 years or older. While patient groups hailed the news, the price of these products provoked sticker shock: Zolgensma costs $2.1 million;

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Fonte: Nature Biotechnology | VOL 37 | JULY 2019 | 697 | www.nature.com/naturebiotechnology