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Besselink-Lobanova A, Maandag NJ, Voermans NC, van der Heijden HF, van der Hoeven JG, Heunks LM

Marking its place as the fourth ALS treatment and pioneering the way as the inaugural gene therapy to gain accelerated approval anchored in biomarker evidence, tofersen is a landmark in the field of gene therapy for ALS (45)

Zygosity Because a homozygous mutant is often more deleterious than a heterozygous mutant, zygosity plays an important role as an indicator

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In cholangiocarcinoma (CCA), the risk of VTE and the role of the tumor cell in the abnormal activation of the blood coagulation cascade are underestimated

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