From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis

Willeke van Roon-Mom^{1

2}, Chantal Ferguson³, Annemieke Aartsma-Rus^{1

2}

Affiliations

¹ Department of Human Genetics, Dutch Center for RNA Therapeutics, Leiden University Medical Center, Leiden, The Netherlands.
² Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
³ RNA Therapeutics Institute, UMass Chan Medical School, Worcester, Massachusetts, USA.

Willeke van Roon-Mom et al. Nucleic Acid Ther. 2023 Aug.

. 2023 Aug;33(4):234-237.

doi: 10.1089/nat.2023.0027.

Willeke van Roon-Mom^{1

2}, Chantal Ferguson³, Annemieke Aartsma-Rus^{1

2}

¹ Department of Human Genetics, Dutch Center for RNA Therapeutics, Leiden University Medical Center, Leiden, The Netherlands.
² Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
³ RNA Therapeutics Institute, UMass Chan Medical School, Worcester, Massachusetts, USA.

No abstract available

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