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The goal of therapy is to stop the acute attack as quickly as possible while providing supportive and symptomatic care

Targeted overexpression of androgen receptor with a liver-specific promoter in transgenic mice

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)

Metabolism 36 , 115121 (1987)

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