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 <i>SOD1</i> Mutated Amyotrophic Lateral Sclerosis-Reference-Cited by-同舟云学术

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

Published:2023-08-01 Issue:4 Volume:33 Page:234-237
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

van Roon-Mom Willeke¹²^ORCID,Ferguson Chantal³,Aartsma-Rus Annemieke¹²^ORCID

Affiliation:

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.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2023.0027

Reference19 articles.

1. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

2. FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

3. The Future of Exon Skipping for Duchenne Muscular Dystrophy

4. Phenotypic variability in amyotrophic lateral sclerosis

5. ALS motor phenotype heterogeneity, focality, and spread: Deconstructing motor neuron degeneration

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