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Metabolism 36 , 115121 (1987)

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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)

What the largest new study found (and why it matters) A nationwide Swedish study followed 2.48 million children (19952019) and used sibling-control analyses a powerful way to account for shared family genetics and environment