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

The Future of Exon Skipping for Duchenne Muscular Dystrophy

(2023) Annemieke Aartsma-Rus   HUMAN GENE THERAPY

A single-cell map of antisense oligonucleotide activity in the brain

(2023) Meredith A Mortberg et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

(2022) Vladislav A. Korobeynikov et al.   NATURE MEDICINE

Huntington’s disease clinical trials corner: April 2022

(2022) Carlos Estevez-Fraga et al.   Journal of Huntingtons Disease

Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities

(2022) Sarah J Tabrizi et al.   LANCET NEUROLOGY

Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

(2022) Timothy M. Miller et al.   NEW ENGLAND JOURNAL OF MEDICINE

Failure of genetic therapies for Huntington’s devastates community

(2021) Diana Kwon   NATURE

Phenotypic variability in amyotrophic lateral sclerosis

(2021) P. Couratier et al.   REVUE NEUROLOGIQUE

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

(2020) Timothy Miller et al.   NEW ENGLAND JOURNAL OF MEDICINE

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

(2018) Alex McCampbell et al.   JOURNAL OF CLINICAL INVESTIGATION

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2017) Annemieke Aartsma-Rus   Nucleic Acid Therapeutics

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

State of play in amyotrophic lateral sclerosis genetics

(2013) Alan E Renton et al.   NATURE NEUROSCIENCE

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

(2009) J. M. Ravits et al.   NEUROLOGY