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

Nucleic Acid TherapeuticsVol. 33, No. 4 Issues in DevelopmentFrom 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 SclerosisWilleke van Roon-Mom, Chantal Ferguson, and Annemieke Aartsma-RusWilleke van Roon-Momhttps://orcid.org/0000-0002-3035-0533Department of Human Genetics, Dutch Center for RNA Therapeutics, Leiden University Medical Center, Leiden, The Netherlands.Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.Search for more papers by this author, Chantal FergusonRNA Therapeutics Institute, UMass Chan Medical School, Worcester, Massachusetts, USA.Search for more papers by this author, and Annemieke Aartsma-RusAddress correspondence to: Annemieke Aartsma-Rus, PhD, Department of Human Genetics, Leiden University Medical Center, Leiden 2333 ZA, The Netherlands E-mail Address: [email protected]https://orcid.org/0000-0003-1565-654XDepartment of Human Genetics, Dutch Center for RNA Therapeutics, Leiden University Medical Center, Leiden, The Netherlands.Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.Search for more papers by this authorPublished Online:9 Aug 2023https://doi.org/10.1089/nat.2023.0027AboutSectionsView articleView Full TextPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail View articleFiguresReferencesRelatedDetails Volume 33Issue 4Aug 2023 InformationCopyright 2023, Mary Ann Liebert, Inc., publishersTo cite this article:Willeke van Roon-Mom, Chantal Ferguson, and Annemieke Aartsma-Rus.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.Nucleic Acid Therapeutics.Aug 2023.234-237.http://doi.org/10.1089/nat.2023.0027Published in Volume: 33 Issue 4: August 9, 2023PDF download